References

Huntington’s disease clinical research congress, October 11-13, 2025, Nashville, TN

2025 · External reference

From antisense RNA to RNA modification: therapeutic potential of RNA-based technologies

10.3390/biomedicines9050550 · 2021 · External reference

Mapping the structural determinants required for AAVrh. 10 transport across the blood-brain barrier

10.1016/j.ymthe.2017.10.017 · 2018 · External reference

CRISPR-Cas systems in neurological diseases

2026 · External reference

DTMP-prime: a deep transformer-based model for predicting prime editing efficiency and PegRNA activity

10.1016/j.omtn.2024.102370 · 2024 · External reference

Mechanistic insights, treatment paradigms, and clinical progress in neurological disorders: current and future prospects

10.3390/ijms24021340 · 2023 · External reference

Current updates of CRISPR/Cas system and anti-CRISPR proteins: innovative applications to improve the genome editing strategies

10.2147/ijn.s479068 · 2024 · External reference

Focused ultrasound enhances transgene expression of intranasal hGDNF DNA nanoparticles in the sonicated brain regions

10.1016/j.jconrel.2023.04.041 · 2023 · External reference

Antisense oligonucleotide therapy for the nervous system: from bench to bedside with emphasis on pediatric neurology

10.3390/pharmaceutics14112389 · 2022 · External reference

Convection-enhanced delivery in children: techniques and applications

10.1007/978-3-030-99166-1_6 · 2022 · External reference

Understanding the potential of genome editing in Parkinson’s disease

10.3390/ijms22179241 · 2021 · External reference

Progress in pseudotyping lentiviral vectors towards cell-specific gene delivery in vivo

10.3390/v17060802 · 2025 · External reference

Immunogenicity of recombinant adeno-associated virus (AAV) vectors for gene transfer

10.1007/s40259-023-00585-7 · 2023 · External reference

The I-CreI meganuclease and its engineered derivatives: applications from cell modification to gene therapy

10.1093/protein/gzq083 · 2011 · External reference

In vitro and in vivo optimization of liposomal nanoparticles based brain targeted vgf gene therapy

10.1016/j.ijpharm.2021.121095 · 2021 · External reference

Unresolved reference

2023 · External reference

Inorganic nanoparticles-based drug delivery systems for neurodegenerative diseases therapy

10.2174/0113816128352935250116064725 · 2025 · External reference

An overview of MR-guided laser interstitial thermal therapy (MRg-LITT) in disrupting the blood-brain barrier: efficacy and duration

10.31083/j.jin2307130 · 2024 · External reference

Correcting a pathogenic mitochondrial DNA mutation by base editing in mice

10.1126/scitranslmed.adr0792 · 2025 · External reference

Polymeric nanoparticles for drug delivery

10.1021/acs.chemrev.3c00705 · 2024 · External reference

TALE and TALEN genome editing technologies

2021 · External reference

Emerging perspectives on prime editor delivery to the brain

10.3390/ph17060763 · 2024 · External reference

RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform

10.1146/annurev.pharmtox.010909.105654 · 2010 · External reference

Microglia-specific IL-10 gene delivery inhibits neuroinflammation and neurodegeneration in a mouse model of Parkinson’s disease

10.1126/scitranslmed.adm8563 · 2024 · External reference

Unresolved reference

2025 · External reference

Fine-tuning of a generative neural network for designing multi-target compounds

10.1007/s10822-021-00392-8 · 2022 · External reference

BBB opening with focused ultrasound in nonhuman primates and Parkinson’s disease patients: targeted AAV vector delivery and PET imaging

10.1126/sciadv.adf4888 · 2023 · External reference

Adeno-associated virus and lentivirus vectors: a refined toolkit for the central nervous system

10.1016/j.coviro.2016.08.004 · 2016 · External reference

A pseudotyped adenovirus serotype 5 vector with serotype 49 fiber knob is an effective vector for vaccine and gene therapy applications

10.1016/j.omtm.2024.101308 · 2024 · External reference

Brain delivery of biotherapeutics via receptor-mediated transcytosis across the blood–brain barrier

10.1039/d5pm00204d · 2025 · External reference

Prime editing of the β1 adrenoceptor in the brain reprograms mouse behavior

2023 · External reference

Therapeutic rAAVrh10 mediated SOD1 silencing in adult SOD1 G93A mice and nonhuman primates

10.1089/hum.2015.122 · 2016 · External reference

Antisense oligonucleotide–mediated MSH3 suppression reduces somatic CAG repeat expansion in Huntington’s disease iPSC–derived striatal neurons

10.1126/scitranslmed.adn4600 · 2025 · External reference

Lipid nanoparticles: versatile drug delivery vehicles for traversing the blood brain barrier to treat brain cancer

10.1002/adfm.202404234 · 2024 · External reference

Polymeric nanoparticles in brain cancer therapy: a review of current approaches

10.3390/polym14142963 · 2022 · External reference

Design, construction and in vitro testing of zinc finger nucleases

10.1038/nprot.2006.231 · 2006 · External reference

Genetic mutations in HSV-1 replication-defective vectors: implications for their safety in gene therapy applications

10.1038/s41434-025-00566-1 · 2025 · External reference

Applications of advanced ASOs, RNA mimics, and others in RNA-based therapeutics

2025 · External reference

Adeno-associated virus toolkit to target diverse brain cells

10.1146/annurev-neuro-111020-100834 · 2022 · External reference

Advanced gene-targeting therapies for motor neuron diseases and muscular dystrophies

10.3390/ijms23094824 · 2022 · External reference

Enhanced CNS transduction from AAV. PHP. eB infusion into the cisterna magna of older adult rats compared to AAV9

10.1038/s41434-021-00244-y · 2022 · External reference

323 TALEN®-based gene edited iPSC-derived NK (iNK) cells demonstrate enhanced antitumor activity

2022 · External reference

Reactive glia-to-GABAergic neuron reprogramming: a “golden touch” strategy to alleviate intractable seizures

10.1186/s42494-022-00104-1 · 2022 · External reference

Gene editing for HD: therapeutic prospects

2024 · External reference

Vector tropism

2023 · External reference

Gene therapy for ocular hypertension using hfCas13d-mediated mRNA targeting

10.1093/pnasnexus/pgaf168 · 2025 · External reference

Unresolved reference

External reference

Receptor ligand-free mesoporous silica nanoparticles: a streamlined strategy for targeted drug delivery across the blood–brain barrier

10.1021/acsnano.3c08993 · 2024 · External reference

The LAGLIDADG homing endonuclease family

2005 · External reference

Strategies for targeted delivery of exosomes to the brain: advantages and challenges

10.3390/pharmaceutics14030672 · 2022 · External reference

Advances in gene therapy techniques to treat LRRK2 gene mutation

10.3390/biom12121814 · 2022 · External reference

Cell reprogramming for regeneration and repair of the nervous system

10.3390/biomedicines10102598 · 2022 · External reference

tRNA therapeutics for genetic diseases

10.1038/s41573-023-00829-9 · 2024 · External reference

Targeting PTB for glia-to-neuron reprogramming in vitro and in vivo for therapeutic development in neurological diseases

10.3390/biomedicines10020399 · 2022 · External reference

Intranasal delivery of nanostructured lipid carriers, solid lipid nanoparticles and nanoemulsions: a current overview of in vivo studies

10.1016/j.apsb.2021.02.012 · 2021 · External reference

RNA editing with CRISPR-Cas13

10.1126/science.aaq0180 · 2017 · External reference

Helper-dependent adenovirus vectors: their use as a gene delivery system to neurons

10.1038/sj.gt.3301208 · 2000 · External reference

Spatiotemporal and genetic regulation of A-to-I editing throughout human brain development

10.1016/j.celrep.2022.111585 · 2022 · External reference

Review of CRISPR/Cas9 sgRNA design tools

10.1007/s12539-018-0298-z · 2018 · External reference

Health research in the era of artificial intelligence: advances in gene-editing study

10.1016/j.medp.2024.100027 · 2024 · External reference

Mesoporous silica nanoparticles for targeted Aβ42 imaging and therapy in traumatic brain injury: enhanced blood–brain barrier penetration and sustained drug delivery

10.1021/acsanm.5c00997 · 2025 · External reference

Optimizing sgRNA structure to improve CRISPR-Cas9 knockout efficiency

10.1186/s13059-015-0846-3 · 2015 · External reference

Impairment of lysosome function and autophagy in rare neurodegenerative diseases

10.1016/j.jmb.2020.02.033 · 2020 · External reference

Advancement in CRISPR/Cas9 technology to better understand and treat neurological disorders

10.1007/s10571-022-01242-3 · 2023 · External reference

Neurodevelopmental disorders: 2024 update

2024 · External reference

Exosomes as biomarkers and therapeutic agents in neurodegenerative diseases: current insights and future directions

10.1007/s12035-025-04825-5 · 2025 · External reference

Gene therapies for neurogenetic disorders

10.1016/j.molmed.2025.01.015 · 2025 · External reference

Gene therapy for Dravet syndrome: promises and impact on disease trigger and secondary modifications

10.20517/rdodj.2024.07 · 2024 · External reference

Cell membrane-and vesicle-based bionic nanodrugs: applications in central nervous system diseases and exploration of nasal–cerebral delivery

10.3390/gels11110846 · 2025 · External reference

Advancing genome editing with artificial intelligence: opportunities, challenges, and future directions

10.3389/fbioe.2023.1335901 · 2024 · External reference

Technological advances in the use of viral and non-viral vectors for delivering genetic and non-genetic cargos for cancer therapy

10.1007/s13346-023-01362-3 · 2023 · External reference

Direct neural reprogramming in situ: existing approaches and their optimization

10.1134/s000629792460426x · 2025 · External reference

Key challenges and optimization practices for non-clinical evaluation of AAV based gene therapy for central nervous system disease

10.1007/s11095-025-03931-2 · 2025 · External reference

Meta-analysis of genetic regulation of RNA editing in the human brain identifies new genes underlying neurological disease

2025 · External reference

RNA-based therapies for neurodevelopmental disorders: innovative tools for molecular correction

10.3389/fmolb.2025.1681647 · 2025 · External reference

Autophagy–lysosome pathway dysfunction in neurodegeneration and cancer: mechanisms and therapeutic opportunities

10.3390/ijms27010366 · 2025 · External reference

The potential of gene editing for Huntington’s disease

10.1016/j.tins.2023.02.005 · 2023 · External reference

Targeted gene therapy of xeroderma pigmentosum cells using meganuclease and TALEN™

10.1371/journal.pone.0078678 · 2013 · External reference

Chloroplast ribosomal intron of Chlamydomonas reinhardtii: in vitro self‐splicing, DNA endonuclease activity and in vivo mobility

10.1002/j.1460-2075.1991.tb04913.x · 1991 · External reference

Linking metabolic syndrome to neurodegeneration mechanisms and potential treatments

10.1007/s12035-025-04947-w · 2025 · External reference

Evaluation of Cas13d as a tool for genetic interaction mapping

10.1038/s41467-025-56747-4 · 2025 · External reference

Direct convective delivery for nervous system gene therapy

10.1016/j.nec.2024.09.002 · 2025 · External reference

Efficient silencing of gene expression with modular trimeric Pol II expression cassettes comprising microRNA shuttles

10.1093/nar/gkp446 · 2009 · External reference

Tofersen for SOD1 ALS

10.1080/17582024.2024.2402216 · 2024 · External reference

Adeno-associated viral vectors as versatile tools for Parkinson’s research, both for disease modeling purposes and for therapeutic uses

10.3390/ijms22126389 · 2021 · External reference

Potential common pathogenesis of several neurodegenerative diseases

10.4103/nrr.nrr-d-24-01054 · 2026 · External reference

Understanding drug nanocarrier and blood–brain barrier interaction based on a microfluidic microphysiological model

10.1039/d2lc01077a · 2023 · External reference

Electroporation-based therapy for brain tumors: a review

10.1115/1.4051184 · 2021 · External reference

CpG-depleted adeno-associated virus vectors evade immune detection

10.1172/jci68205 · 2013 · External reference

Nose-to-brain siRNA delivery by PEI/PPI-based nanoparticles reduces α-synuclein expression in a Parkinson’s disease mouse model

10.1016/j.omtn.2025.102671 · 2025 · External reference

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

10.1016/s0140-6736(16)31408-8 · 2016 · External reference

A multifaceted approach to optimizing AAV delivery to the brain for the treatment of neurodegenerative diseases

10.3389/fnins.2021.747726 · 2021 · External reference

Gene therapy for rare neurological disorders

10.1002/cpt.2543 · 2022 · External reference

Neurodegenerative disorders: mechanisms of degeneration and therapeutic approaches with their clinical relevance

10.1016/j.arr.2024.102357 · 2024 · External reference

Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo

10.1038/s41591-018-0165-9 · 2018 · External reference

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

10.1073/pnas.182412299 · 2002 · External reference

Research progress of gene editing technology in neurological diseases

10.1016/j.gene.2025.149534 · 2025 · External reference

Strategies for enhanced gene delivery to the central nervous system

10.1039/d3na01125a · 2024 · External reference

Advances and challenges in gene therapy for neurodegenerative diseases: a systematic review

10.3390/ijms252312485 · 2024 · External reference

Effects of low-intensity pulsed ultrasound-induced blood–brain barrier opening in P301S mice modeling Alzheimer’s disease tauopathies

10.3390/ijms241512411 · 2023 · External reference

RNA interference (RNAi)-based therapeutics for treatment of rare neurologic diseases

10.1016/j.mam.2022.101148 · 2023 · External reference

Synthetic zinc finger proteins: the advent of targeted gene regulation and genome modification technologies

10.1021/ar500039w · 2014 · External reference

Neurodegenerative disease: models, mechanisms, and a new hope

10.1242/dmm.030205 · 2017 · External reference

Engineering the delivery system for CRISPR-based genome editing

10.1016/j.tibtech.2017.11.006 · 2018 · External reference

Chemical synthesis and biological application of modified oligonucleotides

10.1021/acs.bioconjchem.0c00060 · 2020 · External reference

The promise of epigenetic editing for treating brain disorders

2025 · External reference

Naringenin: a prospective therapeutic agent for Alzheimer’s and Parkinson’s disease

10.1111/jfbc.14415 · 2022 · External reference

CRISPR/Cas13-based approaches for ultrasensitive and specific detection of microRNAs

10.3390/cells10071655 · 2021 · External reference

Off-target effects in CRISPR/Cas9 gene editing

10.3389/fbioe.2023.1143157 · 2023 · External reference

Angiopep-2-modified nanoparticles for brain-directed delivery of therapeutics: a review

10.3390/polym14040712 · 2022 · External reference

Four decades of adenovirus gene transfer vectors: history and current use

10.1016/j.ymthe.2025.03.062 · 2025 · External reference

Molecularly defined hippocampal inputs regulate population dynamics in the prelimbic cortex to suppress context fear memory retrieval

10.1016/j.biopsych.2020.04.014 · 2020 · External reference

Engineering a streamlined virus-like particle for programmable tissue-specific gene delivery

10.1038/s41467-025-64181-9 · 2025 · External reference

Gene silencing therapies for Huntington’s disease

10.61882/nl.4.1.69 · 2025 · External reference

Recent progress in delivery of therapeutic and imaging agents utilizing organic-inorganic hybrid nanoparticles

10.2174/1567201814666171120114034 · 2018 · External reference

Crossing the blood–brain barrier: nanoparticle-based strategies for neurodegenerative disease therapy

10.1007/s13346-025-01887-9 · 2025 · External reference

Injectable hydrogels in central nervous system: unique and novel platforms for promoting extracellular matrix remodeling and tissue engineering

2023 · External reference

Exosomal microRNAs in common mental disorders: mechanisms, biomarker potential and therapeutic implications

10.5498/wjp.v15.i8.108933 · 2025 · External reference

Breaking barriers in Huntington’s disease therapy: focused ultrasound for targeted drug delivery

10.1007/s11064-024-04302-w · 2025 · External reference

Clinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseases

10.1080/15476286.2022.2066334 · 2022 · External reference

Mitochondrial DNA editing: key to the treatment of neurodegenerative diseases

2025 · External reference

Role of oxidative stress in neurodegenerative disorders: a review of reactive oxygen species and prevention by antioxidants

10.1093/braincomms/fcad356 · 2024 · External reference

Immunity in neuromodulation: probing neural and immune pathways in brain disorders

10.1186/s12974-025-03440-4 · 2025 · External reference

Nonsense-mediated mRNA decay in neuronal physiology and neurodegeneration

10.1016/j.tins.2023.07.001 · 2023 · External reference

Opto-CRISPR: new prospects for gene editing and regulation

2025 · External reference

Construction and optimization of herpes simplex virus vectors for central nervous system gene delivery based on CRISPR/Cas9-mediated genome editing

2022 · External reference

Maximizing lentiviral vector gene transfer in the CNS

10.1038/s41434-020-0172-6 · 2021 · External reference

Recent applications of mesoporous silica nanoparticles in gene therapy

10.1002/adhm.202404781 · 2025 · External reference

Targeting miRNA by CRISPR/Cas in cancer: advantages and challenges

2023 · External reference

Precise editing of pathogenic nucleotide repeat expansions in iPSCs using paired prime editor

10.1093/nar/gkae310 · 2024 · External reference

Fine-tuning of the PPR protein directs the RNA editing activity toward C-to-U or U-to-C conversion

10.1038/s41598-025-90722-9 · 2025 · External reference

Next-generation replication-defective HSV vectors for delivery of large DNA payloads

10.1016/j.ymthe.2025.03.055 · 2025 · External reference

CRISPR-Cas9-edited SNCA knockout human induced pluripotent stem cell-derived dopaminergic neurons and their vulnerability to neurotoxicity

10.1248/bpb.b22-00839 · 2023 · External reference

Gene therapy, a novel therapeutic tool for neurological disorders: current progress, challenges and future prospective

10.2174/1566523220999200716111502 · 2020 · External reference

Advances in neurodegenerative disease therapy: stem cell clinical trials and promise of engineered exosomes

10.1111/cns.70577 · 2025 · External reference

Peptide-mediated drug delivery across the blood-brain barrier for targeting brain tumors

10.1080/17425247.2019.1614911 · 2019 · External reference

Lipid nanoparticles: promising treatment approach for Parkinson’s disease

10.3390/ijms23169361 · 2022 · External reference

Biomimetic nanovesicles as a dual gene delivery system for the synergistic gene therapy of Alzheimer’s disease

10.1021/acsnano.3c13150 · 2024 · External reference

Regulatory T cells in neurological disorders and tissue regeneration: mechanisms of action and therapeutic potentials

10.4103/nrr.nrr-d-24-01363 · 2026 · External reference

ADAR-mediated RNA editing of DNA: RNA hybrids is required for DNA double strand break repair

10.1038/s41467-021-25790-2 · 2021 · External reference

CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis

10.1126/sciadv.aax1210 · 2019 · External reference

MRI-guided focused ultrasound

2013 · External reference

Development of efficient AAV2/DJ-based viral vectors to selectively downregulate the expression of neuronal or astrocytic target proteins in the rat central nervous system

10.3389/fnmol.2019.00201 · 2019 · External reference

7 Advances in therapeutic oligonucleotide

10.1515/9783111425085-007 · 2025 · External reference

Gene therapy of neurological and non-neurological diseases using herpes simplex virus vectors

2024 · External reference

Saccharomyces cerevisiae MutLα is a mismatch repair endonuclease

10.1074/jbc.m707617200 · 2007 · External reference

Unresolved reference

External reference

Off-target effects in CRISPR-Cas genome editing for human therapeutics: progress and challenges

2025 · External reference

CRISPR-Cas9 DNA base-editing and prime-editing

10.3390/ijms21176240 · 2020 · External reference

A comprehensive review on the pathophysiology, diagnosis, and emerging therapies in neurodegenerative diseases

2025 · External reference

Recommendations for detection, validation, and evaluation of RNA editing events in cardiovascular and neurological/neurodegenerative diseases

10.1016/j.omtn.2023.102085 · 2024 · External reference

AI-powered predictive modelling for disease diagnostics

2024 · External reference

Engineering microglial exosome-mediated microRNA-124-3p delivery for Alzheimer’s disease combinational therapy

10.1039/d5bm01080b · 2026 · External reference

Induction of antigen-specific tolerance by hepatic AAV immunotherapy regardless of T cell epitope usage or mouse strain background

10.1016/j.omtm.2022.12.011 · 2023 · External reference

Gene therapy-induced antigen-specific tregs inhibit neuro-inflammation and reverse disease in a mouse model of multiple sclerosis

10.1016/j.ymthe.2017.09.001 · 2018 · External reference

AAV-mediated gene delivery of BDNF or GDNF is neuroprotective in a model of Huntington disease

10.1016/j.ymthe.2004.02.016 · 2004 · External reference

Gene editing tool for neurodegenerative diseases

2024 · External reference

Structural basis of Cas3 activation in type IC CRISPR-Cas system

10.1093/nar/gkae723 · 2024 · External reference

Revolutionizing CRISPR technology with artificial intelligence

10.1038/s12276-025-01462-9 · 2025 · External reference

Advances of genome editing with CRISPR/Cas9 in neurodegeneration: the right path towards therapy

10.3390/biomedicines11123333 · 2023 · External reference

Transposon-encoded CRISPR–Cas systems direct RNA-guided DNA integration

10.1038/s41586-019-1323-z · 2019 · External reference

Transcriptome engineering with RNA-targeting type VI-D CRISPR effectors

10.1016/j.cell.2018.02.033 · 2018 · External reference

CRISPR in mobile genetic elements: counter-defense, inter-element competition and RNA-guided transposition

10.1186/s12915-024-02090-x · 2024 · External reference

Applications of the versatile CRISPR‐Cas13 RNA targeting system

10.1002/wrna.1694 · 2022 · External reference

Spatial localization immobilization of metals and enzymes at Pickering emulsion droplet interfaces for chemo-enzymatic cascade catalysis

2024 · External reference

Novel perspective of therapeutic modules to overcome motor and nonmotor symptoms in Parkinson’s disease

10.3934/neuroscience.2024020 · 2024 · External reference

Therapeutic potential of genome editing tools in neurodegenerative diseases

2025 · External reference

Neuroinflammation and neurodegenerative diseases

2025 · External reference

Brain nucleic acid delivery and genome editing via focused ultrasound-mediated blood–brain barrier opening and long-circulating nanoparticles

10.1021/acsnano.4c05270 · 2024 · External reference

Non-replicative herpes simplex virus genomic and amplicon vectors for gene therapy-an update

10.1038/s41434-024-00500-x · 2025 · External reference

Unresolved reference

External reference

Retrospective review of intra-cerebrospinal fluid (CSF) drug delivery in CNS malignancies: safety, clinical efficacy and pharmacokinetic profiles of intracerebroventricular (ICV), lumbar intrathecal (LIT), and intra-cisterna magna (ICM) Injections

10.3390/cancers17081263 · 2025 · External reference

Prime editing with genuine Cas9 nickases minimizes unwanted indels

10.1038/s41467-023-37507-8 · 2023 · External reference

Nanodrug delivery systems for regulating microglial polarization in ischemic stroke treatment: a review

10.1177/20417314241237052 · 2024 · External reference

Gene therapy in ALS and SMA: advances, challenges and perspectives

10.3390/ijms24021130 · 2023 · External reference

Modulation of engineered nanomaterial interactions with organ barriers for enhanced drug transport

10.1039/d1cs00574j · 2023 · External reference

Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial

10.1038/s41591-025-03919-w · 2025 · External reference

Systematic identification of abundant A-to-I editing sites in the human transcriptome

10.1038/nbt996 · 2004 · External reference

Gene and RNA editing: revolutionary approaches to treating diseases

10.1002/mco2.70389 · 2025 · External reference

Plant base editing and prime editing: the current status and future perspectives

10.1111/jipb.13425 · 2023 · External reference

RNA editing underlies genetic risk of common inflammatory diseases

10.1038/s41586-022-05052-x · 2022 · External reference

CRISPR/Cas9 therapeutics: progress and prospects

10.1038/s41392-023-01309-7 · 2023 · External reference

CRISPR base editing of cis-regulatory elements enables the perturbation of neurodegeneration-linked genes

10.1016/j.ymthe.2022.08.008 · 2022 · External reference

Crossing the blood-brain barrier with AAV vectors

10.1007/s11011-020-00630-2 · 2021 · External reference

Tet1 peptide and zinc (II)-adenine multifunctional module functionalized polycations as efficient siRNA carriers for Parkinson’s disease

10.1016/j.jconrel.2024.01.042 · 2024 · External reference

Genomic and transcriptomic approaches advance the diagnosis and prognosis of neurodegenerative diseases

10.3390/genes16020135 · 2025 · External reference

Serious neurologic adverse events in tofersen clinical trials for amyotrophic lateral sclerosis

10.1002/mus.28372 · 2025 · External reference

Rational design of base, sugar and backbone modifications improves ADAR-mediated RNA editing

10.1093/nar/gkae681 · 2024 · External reference

Engineered transfer RNAs for suppression of premature termination codons

10.1038/s41467-019-08329-4 · 2019 · External reference

Two high-fidelity variants: efSaCas9 and SaCas9-HF, which one is better?

10.1038/s41434-022-00319-4 · 2022 · External reference

New advances in using virus-like particles and related technologies for eukaryotic genome editing delivery

10.3390/ijms23158750 · 2022 · External reference

Synapsin 1 promotes Aβ generation via BACE1 modulation

10.1371/journal.pone.0226368 · 2019 · External reference

Superparamagnetic iron oxide nanoparticles (SPIONs) in targeting brain tumors: advances and challenges

10.1007/s12032-025-02913-z · 2025 · External reference

Therapeutic potential of luteolin in neurodegenerative disorders: targeting Nrf2, NFĸB, MAPK, and JAK-STAT pathways to combat neuroinflammation and apoptosis

10.1007/s10787-025-01846-3 · 2025 · External reference

Autophagic and endo-lysosomal dysfunction in neurodegenerative disease

10.1186/s13041-019-0504-x · 2019 · External reference

Emerging trends in virus and virus-like particle gene therapy delivery to the brain

10.1016/j.omtn.2024.102280 · 2024 · External reference

Comparison of three computational tools for the prediction of RNA tertiary structures

10.3390/ncrna10060055 · 2024 · External reference

Epigenetic editing in neurological and neuropsychiatric disorders: pioneering next-gen therapeutics for precision gene control

10.1007/s12035-025-05590-1 · 2026 · External reference

Gene therapy for neurodegenerative diseases: slowing down the ticking clock

10.3389/fnins.2020.580179 · 2020 · External reference

CNS transduction benefits of AAV-PHP. eB over AAV9 are dependent on administration route and mouse strain

10.1016/j.omtm.2020.10.011 · 2020 · External reference

Predicting prime editing efficiency and product purity by deep learning

10.1038/s41587-022-01613-7 · 2023 · External reference

CRISPR-CAS9 D10A nickase target-specific fluorescent labeling of double strand DNA for whole genome mapping and structural variation analysis

10.1093/nar/gkv878 · 2016 · External reference

Widespread neuron-specific transgene expression in brain and spinal cord following synapsin promoter-driven AAV9 neonatal intracerebroventricular injection

10.1016/j.neulet.2014.05.044 · 2014 · External reference

Genome editing in iPSC-based neural systems: from disease models to future therapeutic strategies

10.3389/fgeed.2021.630600 · 2021 · External reference

Novel focused ultrasound gene therapy approach noninvasively restores dopaminergic neuron function in a rat Parkinson’s disease model

10.1021/acs.nanolett.7b00616 · 2017 · External reference

Single-dose gene-replacement therapy for spinal muscular atrophy

10.1056/nejmoa1706198 · 2017 · External reference

Chemical modifications in RNA: elucidating the chemistry of dsRNA-specific adenosine deaminases (ADARs)

10.1021/acs.accounts.3c00390 · 2023 · External reference

Generation of human induced pluripotent stem cell-derived bona fide neural stem cells for ex vivo gene therapy of metachromatic leukodystrophy

10.5966/sctm.2015-0414 · 2017 · External reference

Blood–brain barrier opening of the default mode network in Alzheimer’s disease with magnetic resonance-guided focused ultrasound

10.1093/brain/awac459 · 2023 · External reference

Current progress in magnetic resonance-guided focused ultrasound to facilitate drug delivery across the blood-brain barrier

10.3390/pharmaceutics16060719 · 2024 · External reference

SPARCLE, a p53-induced lncRNA, controls apoptosis after genotoxic stress by promoting PARP-1 cleavage

10.1016/j.molcel.2022.01.001 · 2022 · External reference

Microglia promote inflammatory cell death upon neuronal mitochondrial impairment during neurodegeneration

10.1038/s41594-025-01602-9 · 2025 · External reference

Comparative analysis of the capsid structures of AAVrh. 10, AAVrh. 39, and AAV8

10.1128/jvi.01769-19 · 2020 · External reference

Structural study of Aavrh. 10 receptor and antibody interactions

10.1128/jvi.01249-21 · 2021 · External reference

Battling neurodegenerative diseases with adeno-associated virus-based approaches

10.3390/v12040460 · 2020 · External reference

Genetic and epigenetic determinants of reactivation of Mecp2 and the inactive X chromosome in neural stem cells

10.1016/j.stemcr.2022.01.008 · 2022 · External reference

Polysorbate emulsifier in drug delivery systems for cancer and neurodegenerative diseases: current status and emerging applications

2025 · External reference

TOMM40 and APOE gene expression and cognitive decline in Japanese Alzheimer’s disease subjects

10.3233/jad-170361 · 2017 · External reference

Advances in CRISPER/Cas system and genome editing technologies for the treatment of neurodegenerative diseases

2025 · External reference

SPLICER: a highly efficient base editing toolbox that enables in vivo therapeutic exon skipping

10.1038/s41467-024-54529-y · 2024 · External reference

Durable tissue-specific transgene expression in newborn mice following intraperitoneal delivery of non-cytotoxic HSV vectors

10.1016/j.omtm.2025.101573 · 2025 · External reference

Aptamer functionalization of nanosystems for glioblastoma targeting through the blood–brain barrier

10.1021/acs.jmedchem.7b00527 · 2017 · External reference

An RNA-targeting CRISPR–Cas13d system alleviates disease-related phenotypes in Huntington’s disease models

10.1038/s41593-022-01207-1 · 2023 · External reference

Development and characterization of a vector system based on the simian adenovirus type 25

2023 · External reference

Controlling the expression level of the neuronal reprogramming factors for a successful reprogramming outcome

10.3390/cells13141223 · 2024 · External reference

Tumor-derived extracellular vesicles disrupt the blood–brain barrier endothelium following high-frequency irreversible electroporation

10.1038/s41598-024-79019-5 · 2024 · External reference

Nucleic acid therapeutics: past, present, and future

10.1016/j.omtn.2024.102440 · 2025 · External reference

Unresolved reference

2024 · External reference

Blood brain barrier permeable gold nanocluster for targeted brain imaging and therapy: an in vitro and in vivo study

10.1039/c7tb02247f · 2017 · External reference

Non-invasive delivery of stealth, brain-penetrating nanoparticles across the blood− brain barrier using MRI-guided focused ultrasound

10.1016/j.jconrel.2014.06.031 · 2014 · External reference

A review: computational approaches to design sgRNA of CRISPR-Cas9

10.2174/1574893616666210601105553 · 2022 · External reference

Post-transcriptional regulation of the transcriptional apparatus in neuronal development

10.3389/fnmol.2024.1483901 · 2024 · External reference

Rapid CRISPR–Cas9 target-strand nicking can provide phage resistance by reducing DNA abundance

10.1093/nar/gkaf900 · 2025 · External reference

Applications and developments of gene therapy drug delivery systems for neurological disorders

2024 · External reference

The role of metals in the neuroregenerative action of BDNF, GDNF, NGF and other neurotrophic factors

10.3390/biom12081015 · 2022 · External reference

Mechanisms of autophagy–lysosome dysfunction in neurodegenerative diseases

10.1038/s41580-024-00757-5 · 2024 · External reference

Gene silencing and editing strategies for neurodegenerative diseases

2018 · External reference

Gene therapy strategies: gene augmentation

2020 · External reference

Biomarkers and therapeutic strategies targeting microglia in neurodegenerative diseases: current status and future directions

10.1186/s13024-025-00867-4 · 2025 · External reference

Neurotrophins and other growth factors in the pathogenesis of Alzheimer’s disease

10.3390/life13030647 · 2023 · External reference

10.21203/rs.3.rs-4619764/v1

10.21203/rs.3.rs-4619764/v1 · External reference

DNA targeting by compact Cas9d and its resurrected ancestor

10.1038/s41467-024-55573-4 · 2025 · External reference

Robust axonal regeneration occurs in the injured CAST/Ei mouse CNS

10.1016/j.neuron.2015.05.005 · 2015 · External reference

Unresolved reference

External reference

Autophagic/lysosomal dysfunction in Alzheimer’s disease

10.1186/alzrt217 · 2013 · External reference

From lab bench to hope: emerging gene therapies in clinical trials for Alzheimer’s disease

10.1007/s12035-024-04285-3 · 2025 · External reference

Advances in neuronal regeneration: hydrogel-based delivery systems loaded with extracellular vesicles in modulating neural impulses and tissue repair

10.1016/j.eurpolymj.2024.113457 · 2024 · External reference

Exploring pro-inflammatory immunological mediators: unraveling the mechanisms of neuroinflammation in lysosomal storage diseases

10.3390/biomedicines11041067 · 2023 · External reference

A miR-124-mediated post-transcriptional mechanism controlling the cell fate switch of astrocytes to induced neurons

10.1016/j.stemcr.2023.02.009 · 2023 · External reference

Post-transcriptional mechanisms controlling neurogenesis and direct neuronal reprogramming

10.4103/1673-5374.390976 · 2024 · External reference

Gene therapy approach with an emphasis on growth factors: theoretical and clinical outcomes in neurodegenerative diseases

10.1007/s12035-021-02555-y · 2022 · External reference

Brain gene therapy with Trojan horse lipid nanoparticles

10.1016/j.molmed.2023.02.004 · 2023 · External reference

Onasemnogene abeparvovec in type 1 spinal muscular atrophy: a systematic review and meta-analysis

10.1089/hum.2022.161 · 2023 · External reference

CRISPR-guided epigenetic modulation as a next-generation therapeutic strategy in chronic inflammatory diseases

2025 · External reference

Neuromodulator signaling bidirectionally controls vesicle numbers in human synapses

10.1016/j.cell.2019.09.011 · 2019 · External reference

An optimized SpCas9 high-fidelity variant for direct protein delivery

10.1016/j.ymthe.2023.03.007 · 2023 · External reference

Neuron-derived extracellular vesicles modulate microglia activation and function

10.3390/biology10100948 · 2021 · External reference

Viral-mediated gene therapy in pediatric neurological disorders

10.1007/s12519-022-00669-4 · 2024 · External reference

A c.726C>G (p. Tyr242Ter) nonsense mutation-associated with splicing alteration (NASA) of WDR45 gene underlies β-propeller protein-associated neurodegeneration (BPAN)

10.1016/j.heliyon.2024.e30438 · 2024 · External reference

Management of neuroinflammatory responses to AAV-mediated gene therapies for neurodegenerative diseases

10.3390/brainsci10020119 · 2020 · External reference

Recruitment of CRISPR-Cas systems by Tn7-like transposons

10.1073/pnas.1709035114 · 2017 · External reference

Genome editing in plants via designed zinc finger nucleases

10.1007/s11627-015-9663-3 · 2015 · External reference

Therapeutic promise of engineered nonsense suppressor tRNAs

10.1002/wrna.1641 · 2021 · External reference

Immune responses and immunosuppressive strategies for adeno-associated virus-based gene therapy for treatment of central nervous system disorders: current knowledge and approaches

10.1089/hum.2022.138 · 2022 · External reference

Light-induced expression of gRNA allows for optogenetic gene editing of T lymphocytes in vivo

10.1093/nar/gkaf213 · 2025 · External reference

Systems of adenoviral gene transfer vectors moving into their fourth generation

2025 · External reference

Decoding single-cell molecular mechanisms in astrocyte-to-iN reprogramming via Ngn2-and Pax6-mediated direct lineage switching

10.1186/s40001-024-01989-z · 2024 · External reference

CRISPR-based genome-editing tools for Huntington’s disease research and therapy

10.1007/s12264-022-00880-3 · 2022 · External reference

CRISPR-Cas9-based technology and its relevance to gene editing in Parkinson’s disease

10.3390/pharmaceutics14061252 · 2022 · External reference

Emerging clinical applications of ADAR based RNA editing

10.1093/stcltm/szaf016 · 2025 · External reference

Creating designed zinc-finger nucleases with minimal cytotoxicity

10.1016/j.jmb.2010.10.043 · 2011 · External reference

Tackling neurodegenerative diseases: animal models of Alzheimer’s disease and Parkinson’s disease

10.1007/s13258-013-0116-2 · 2013 · External reference

Nose-to-brain delivery of BACE1 siRNA loaded in solid lipid nanoparticles for Alzheimer’s therapy

10.1016/j.colsurfb.2017.01.031 · 2017 · External reference

CLUSTER guide RNAs enable precise and efficient RNA editing with endogenous ADAR enzymes in vivo

10.1038/s41587-021-01105-0 · 2022 · External reference

Mitochondrial dysfunctions, oxidative stress and neuroinflammation as therapeutic targets for neurodegenerative diseases: an update on current advances and impediments

10.1016/j.neubiorev.2022.104961 · 2023 · External reference

Loss of TREM2 rescues hyperactivation of microglia, but not lysosomal deficits and neurotoxicity in models of progranulin deficiency

10.15252/embj.2021109108 · 2022 · External reference

Gap junctions between striatal d1 neurons and cholinergic interneurons

10.3389/fncel.2021.674399 · 2021 · External reference

T-CAST: an optimized CAST-Seq pipeline for TALEN confirms superior safety and efficacy of obligate-heterodimeric scaffolds

10.3389/fgeed.2023.1130736 · 2023 · External reference

High-capacity adenoviral vectors: expanding the scope of gene therapy

10.3390/ijms21103643 · 2020 · External reference

Emerging gene therapies for Alzheimer’s and Parkinson’s diseases: an overview of clinical trials and promising candidates

2024 · External reference

Transgene-free genome editing in citrus and poplar trees using positive and negative selection markers

10.1007/s00299-025-03627-2 · 2025 · External reference

Efficient LRP1-mediated uptake and low cytotoxicity of peptide L57 in vitro shows its promise as CNS drug delivery vector

10.1016/j.xphs.2020.09.019 · 2021 · External reference

Divergent landscapes of A-to-I editing in postmortem and living human brain

10.1038/s41467-024-49268-z · 2024 · External reference

Microglia-specific targeting by novel capsid-modified AAV6 vectors

10.1038/mtm.2016.26 · 2016 · External reference

EAAT2 and the molecular signature of amyotrophic lateral sclerosis

2017 · External reference

Optimized prime editing of the Alzheimer’s disease-associated APOE4 mutation

10.1016/j.stemcr.2024.11.002 · 2025 · External reference

10.20944/preprints202504.0007.v1

10.20944/preprints202504.0007.v1 · External reference

Transgene delivery system: viral, nonviral, and other methods for central nervous system

2025 · External reference

Synthetic and natural polymers enhancing drug delivery and their treatment: a comprehensive review

10.22270/jddt.v14i10.6802 · 2024 · External reference

CRISPR–Cas9-based genetic engineering for crop improvement under drought stress

10.1080/21655979.2021.1969831 · 2021 · External reference

A review of the development of biopolymer hydrogel-based scaffold materials for drug delivery and tissue engineering applications

10.3390/gels11030178 · 2025 · External reference

Solid lipid nanoparticles (SLNs): an advanced drug delivery system targeting brain through BBB

10.3390/pharmaceutics13081183 · 2021 · External reference

When will RNA get its AlphaFold moment?

10.1093/nar/gkad726 · 2023 · External reference

Optimized design parameters for CRISPR Cas9 and Cas12a homology-directed repair

10.1038/s41598-021-98965-y · 2021 · External reference

Dysregulation of autophagy as a common mechanism in lysosomal storage diseases

10.1042/ebc20170055 · 2017 · External reference

Lysosomal storage diseases as disorders of autophagy

10.4161/auto.5227 · 2008 · External reference

Non-invasive low pulsed electrical fields for inducing BBB disruption in mice—feasibility demonstration

10.3390/pharmaceutics13020169 · 2021 · External reference

Carbon dots as versatile nano-architectures for the treatment of neurological disorders

2025 · External reference

CRISPR/Cas9 gene editing: a new hope for Alzheimer’s disease

2025 · External reference

Implications of AAV serotypes in neurological disorders: current clinical applications and challenges

10.3390/ctn9030032 · 2025 · External reference

Current state of human gene therapy: approved products and vectors

10.3390/ph16101416 · 2023 · External reference

Significant enhancement of adeno‐associated virus vector yield for gene therapy Using multidimensional optimization strategies

10.1096/fj.202502611rr · 2025 · External reference

Targeting transferrin receptor 1 for enhancing drug delivery through the blood–brain barrier for Alzheimer’s disease

10.3390/ijms26199793 · 2025 · External reference

Chemically modified platforms for better RNA therapeutics

10.1021/acs.chemrev.3c00611 · 2024 · External reference

Controlling and enhancing CRISPR systems

10.1038/s41589-020-00700-7 · 2021 · External reference

Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy

10.2174/156652311794520111 · 2011 · External reference

Site-directed RNA repair of endogenous Mecp2 RNA in neurons

10.1073/pnas.1715320114 · 2017 · External reference

Advances in gold nanoparticles for the diagnosis and management of Alzheimer’s disease

10.3390/pharmaceutics17091158 · 2025 · External reference

Nuclear gene targeting in C hlamydomonas using engineered zinc‐finger nucleases

10.1111/tpj.12066 · 2013 · External reference

Recombinant adeno-associated virus vectors for gene therapy of the central nervous system: delivery routes and clinical aspects

10.3390/biomedicines12071523 · 2024 · External reference

Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington’s disease models

10.1093/brain/awae266 · 2024 · External reference

Genome editing using CRISPR, CAST, and Fanzor systems

10.1016/j.mocell.2024.100086 · 2024 · External reference

Design and development of nanomaterial-based drug carriers to overcome the blood–brain barrier by using different transport mechanisms

10.3390/ijms221810118 · 2021 · External reference

Neurotrophic factors in the physiology of motor neurons and their role in the pathobiology and therapeutic approach to amyotrophic lateral sclerosis

10.3389/fnmol.2023.1238453 · 2023 · External reference

Projections for prevalence of Parkinson’s disease and its driving factors in 195 countries and territories to 2050: modelling study of global burden of disease study 2021

10.1136/bmj-2024-080952 · 2025 · External reference

Neuronal enriched extracellular vesicle miR-122-5p as a potential biomarker for Alzheimer’s disease

10.3390/cells14221784 · 2025 · External reference

Emerging genetic therapies in epilepsy

10.1055/a-2544-1530 · 2025 · External reference

Recent developments in gene therapy for Parkinson’s disease

10.1016/j.ymthe.2025.03.030 · 2025 · External reference

Dynamic landscape and regulation of RNA editing in mammals

10.1038/nature24041 · 2017 · External reference

miRNAs: new biomarkers and therapeutic targets in dementia

10.2174/1381612823666170111094702 · 2017 · External reference

A designer AAV variant permits efficient retrograde access to projection neurons

10.1016/j.neuron.2016.09.021 · 2016 · External reference

Targeting non-coding RNAs in neurodegeneration: advances in therapeutic RNA modalities and next-gen delivery technologies

10.2174/0115672050421604251108045622 · 2026 · External reference

Insights into therapeutic approaches for the treatment of neurodegenerative diseases targeting metabolic syndrome

10.1007/s11033-025-10346-0 · 2025 · External reference

using whole exome sequencing to identify genetic causes of neurodevelopmental disorders in a cohort of 11 patients: a single center experience

10.3390/ijms262010176 · 2025 · External reference

Regulatory insights on gene therapies, adeno-associated virus-based gene therapies, cell/tissue-based products, and medical care/practice in cell and gene therapies: report from the 7th Asia partnership conference-April 25, 2024

10.1016/j.reth.2025.02.007 · 2025 · External reference

Mechanism of CRISPER/Cas9-mediated genome editing: scope and opportunities

2021 · External reference

Toward the development of epigenome editing-based therapeutics: potentials and challenges

10.3390/ijms24054778 · 2023 · External reference

Acute exposure to 27.12 MHz radiofrequency electromagnetic field disrupts blood-brain barrier integrity via eNOS activation and occludin down-regulation

2025 · External reference

In vitro CRISPR/Cas9-directed gene editing to model LRRK2 G2019S Parkinson’s disease in common marmosets

10.1038/s41598-020-60273-2 · 2020 · External reference

Recombinant human myelin-associated glycoprotein promoter drives selective AAV-mediated transgene expression in oligodendrocytes

10.3389/fnmol.2016.00013 · 2016 · External reference

Mitochondrial-based therapies for neurodegenerative diseases: a review of the current literature

10.1007/s00210-025-04014-0 · 2025 · External reference

Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?

10.1038/s41434-021-00232-2 · 2021 · External reference

AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice

10.1038/s41586-022-04533-3 · 2022 · External reference

Targeted delivery of hybrid nanovesicles for enhanced brain penetration to achieve synergistic therapy of glioma

10.1016/j.jconrel.2023.11.033 · 2024 · External reference

Recent advances and clinical prospects of non-viral brain-targeted gene delivery systems

10.1063/5.0255745 · 2025 · External reference

Seroprevalence of binding and neutralizing antibodies against 18 adeno-associated virus types in patients with neuromuscular disorders

2024 · External reference

Innate immune response to viral vectors in gene therapy

10.3390/v15091801 · 2023 · External reference

Modifiers of GRN-associated frontotemporal lobar degeneration

10.1016/j.molmed.2017.08.004 · 2017 · External reference

Treating Parkinson’s disease by astrocyte reprogramming: progress and challenges

10.1126/sciadv.abg3198 · 2021 · External reference

RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice

10.1016/j.ymthe.2025.05.010 · 2025 · External reference

Immunology of cell and gene therapy approaches for neurologic diseases

10.1016/b978-0-323-90120-8.00018-6 · 2024 · External reference

Programmable gene insertion in human cells with a laboratory-evolved CRISPR-associated transposase

10.1126/science.adt5199 · 2025 · External reference

Exosomes and biomimetic nanovesicles-mediated anti-glioblastoma therapy: a head-to-head comparison

10.1016/j.jconrel.2021.07.004 · 2021 · External reference

MRI-guided focused ultrasound for treating Parkinson’s disease with human mesenchymal stem cells

10.1038/s41598-025-85811-8 · 2025 · External reference

The blood–brain barrier cell-targeted gene delivery system to enhance nerve growth factor protein secretion in the brain

10.1021/acsbiomaterials.0c01113 · 2020 · External reference

The effect of CpG sequences on capsid-specific CD8+ T cell responses to AAV vector gene transfer

10.1016/j.ymthe.2019.11.014 · 2020 · External reference

In vivo interactions of nucleic acid nanostructures with cells

10.1002/adma.202314232 · 2025 · External reference

Vectors in CRISPR gene editing for neurological disorders: challenges and opportunities

10.1002/adbi.202400374 · 2025 · External reference

Nucleic acid delivery by ionizable nanocarriers for brain disease treatment

10.1002/brx2.7 · 2023 · External reference

Hydrogels in gene delivery techniques for regenerative medicine and tissue engineering

10.1002/mabi.202300577 · 2024 · External reference

An engineered UGA suppressor tRNA gene for disease-agnostic AAV delivery

2026 · External reference

An RNA editing strategy rescues gene duplication in a mouse model of MECP2 duplication syndrome and nonhuman primates

10.1038/s41593-024-01838-6 · 2025 · External reference

Overcoming the blood–brain barrier: advanced strategies in targeted drug delivery for neurodegenerative diseases

10.3390/pharmaceutics17081041 · 2025 · External reference

Non-invasive CRISPR/Cas9 nanocapsules specifically edit α-synuclein for effective Parkinson’s disease treatment

10.1016/j.nantod.2025.102903 · 2026 · External reference

MiR-124 enriched exosomes promoted the M2 polarization of microglia and enhanced hippocampus neurogenesis after traumatic brain injury by inhibiting TLR4 pathway

10.1007/s11064-018-02714-z · 2019 · External reference

Overview of the nonclinical development strategies and class effects of oligonucleotide-based therapeutics

2024 · External reference

Genetically engineered exosomes display RVG peptide and selectively enrich a neprilysin variant: a potential formulation for the treatment of Alzheimer’s disease

10.1080/1061186x.2021.1929257 · 2021 · External reference

In situ direct reprogramming of astrocytes to neurons via polypyrimidine tract-binding protein 1 knockdown in a mouse model of ischemic stroke

10.4103/1673-5374.390957 · 2024 · External reference

A review of mitochondrial dysfunction, pathophysiology and therapeutic prospects in neurodegenerative diseases

10.1186/s13064-025-00223-8 · 2025 · External reference

Immune responses to adeno-associated virus vectors

10.2174/1566523054065039 · 2005 · External reference

TALEN-interceded genome editing in plants: unveiling new frontiers in secondary metabolite improvement and genetic diversity

10.3390/plants14193024 · 2025 · External reference

Applications of gold nanoparticles in brain diseases across the blood-brain barrier

10.2174/0929867329666220527121943 · 2022 · External reference

Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy

2024 · External reference

Hijacking of the nervous system in cancer: mechanism and therapeutic targets

10.1186/s12943-025-02246-5 · 2025 · External reference

Nose to brain: exploring the progress of intranasal delivery of solid lipid nanoparticles and nanostructured lipid carriers

10.2147/ijn.s497480 · 2024 · External reference

A comprehensive review of AAV-mediated strategies targeting microglia for therapeutic intervention of neurodegenerative diseases

10.1186/s12974-024-03232-2 · 2024 · External reference

10.2139/ssrn.6076076

10.2139/ssrn.6076076 · External reference

Lysosomal dysfunction and other pathomechanisms in FTLD: evidence from progranulin genetics and biology

2021 · External reference

Utilizing AI-enhanced multi-omics integration for predictive modeling of disease susceptibility in functional phenotypes

10.53469/jtpes.2024.04(02).07 · 2024 · External reference

Tracking-seq reveals the heterogeneity of off-target effects in CRISPR–Cas9-mediated genome editing

10.1038/s41587-024-02307-y · 2025 · External reference

Gene therapy for neurodegenerative disease: clinical potential and directions

10.3389/fnmol.2021.618171 · 2021 · External reference

Blood-brain barrier–penetrating single CRISPR-Cas9 nanocapsules for effective and safe glioblastoma gene therapy

10.1126/sciadv.abm8011 · 2022 · External reference

Adeno-associated virus vectors: principles, practices, and prospects in gene therapy

10.3390/v17020239 · 2025 · External reference