Abstract
No public abstract is available from the verified source records.
Fatemeh Saadatpour
Abstract
Authors
Institutions
Provenance
crossref
Confidence 100%
openalex
Confidence 95%
datacite
Confidence 0%
No local reference links have been materialized yet.
No local citing links have been materialized yet.
Huntington’s disease clinical research congress, October 11-13, 2025, Nashville, TN
2025
From antisense RNA to RNA modification: therapeutic potential of RNA-based technologies
10.3390/biomedicines9050550 · 2021
Mapping the structural determinants required for AAVrh. 10 transport across the blood-brain barrier
10.1016/j.ymthe.2017.10.017 · 2018
CRISPR-Cas systems in neurological diseases
2026
DTMP-prime: a deep transformer-based model for predicting prime editing efficiency and PegRNA activity
10.1016/j.omtn.2024.102370 · 2024
Mechanistic insights, treatment paradigms, and clinical progress in neurological disorders: current and future prospects
10.3390/ijms24021340 · 2023
Current updates of CRISPR/Cas system and anti-CRISPR proteins: innovative applications to improve the genome editing strategies
10.2147/ijn.s479068 · 2024
Focused ultrasound enhances transgene expression of intranasal hGDNF DNA nanoparticles in the sonicated brain regions
10.1016/j.jconrel.2023.04.041 · 2023
Antisense oligonucleotide therapy for the nervous system: from bench to bedside with emphasis on pediatric neurology
10.3390/pharmaceutics14112389 · 2022
Convection-enhanced delivery in children: techniques and applications
10.1007/978-3-030-99166-1_6 · 2022
Understanding the potential of genome editing in Parkinson’s disease
10.3390/ijms22179241 · 2021
Progress in pseudotyping lentiviral vectors towards cell-specific gene delivery in vivo
10.3390/v17060802 · 2025
Immunogenicity of recombinant adeno-associated virus (AAV) vectors for gene transfer
10.1007/s40259-023-00585-7 · 2023
The I-CreI meganuclease and its engineered derivatives: applications from cell modification to gene therapy
10.1093/protein/gzq083 · 2011
In vitro and in vivo optimization of liposomal nanoparticles based brain targeted vgf gene therapy
10.1016/j.ijpharm.2021.121095 · 2021
Unresolved referenced work
2023
Inorganic nanoparticles-based drug delivery systems for neurodegenerative diseases therapy
10.2174/0113816128352935250116064725 · 2025
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
Correcting a pathogenic mitochondrial DNA mutation by base editing in mice
10.1126/scitranslmed.adr0792 · 2025
Polymeric nanoparticles for drug delivery
10.1021/acs.chemrev.3c00705 · 2024
TALE and TALEN genome editing technologies
2021
Emerging perspectives on prime editor delivery to the brain
10.3390/ph17060763 · 2024
RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform
10.1146/annurev.pharmtox.010909.105654 · 2010
Microglia-specific IL-10 gene delivery inhibits neuroinflammation and neurodegeneration in a mouse model of Parkinson’s disease
10.1126/scitranslmed.adm8563 · 2024
Unresolved referenced work
2025
Fine-tuning of a generative neural network for designing multi-target compounds
10.1007/s10822-021-00392-8 · 2022
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
Adeno-associated virus and lentivirus vectors: a refined toolkit for the central nervous system
10.1016/j.coviro.2016.08.004 · 2016
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
Brain delivery of biotherapeutics via receptor-mediated transcytosis across the blood–brain barrier
10.1039/d5pm00204d · 2025
Prime editing of the β1 adrenoceptor in the brain reprograms mouse behavior
2023
Therapeutic rAAVrh10 mediated SOD1 silencing in adult SOD1 G93A mice and nonhuman primates
10.1089/hum.2015.122 · 2016
Antisense oligonucleotide–mediated MSH3 suppression reduces somatic CAG repeat expansion in Huntington’s disease iPSC–derived striatal neurons
10.1126/scitranslmed.adn4600 · 2025
Lipid nanoparticles: versatile drug delivery vehicles for traversing the blood brain barrier to treat brain cancer
10.1002/adfm.202404234 · 2024
Polymeric nanoparticles in brain cancer therapy: a review of current approaches
10.3390/polym14142963 · 2022
Design, construction and in vitro testing of zinc finger nucleases
10.1038/nprot.2006.231 · 2006
Genetic mutations in HSV-1 replication-defective vectors: implications for their safety in gene therapy applications
10.1038/s41434-025-00566-1 · 2025
Applications of advanced ASOs, RNA mimics, and others in RNA-based therapeutics
2025
Adeno-associated virus toolkit to target diverse brain cells
10.1146/annurev-neuro-111020-100834 · 2022
Advanced gene-targeting therapies for motor neuron diseases and muscular dystrophies
10.3390/ijms23094824 · 2022
Adeno-associated virus vectors: principles, practices, and prospects in gene therapy
10.3390/v17020239 · doi-reference
Blood-brain barrier–penetrating single CRISPR-Cas9 nanocapsules for effective and safe glioblastoma gene therapy
10.1126/sciadv.abm8011 · doi-reference
Gene therapy for neurodegenerative disease: clinical potential and directions
10.3389/fnmol.2021.618171 · doi-reference
Tracking-seq reveals the heterogeneity of off-target effects in CRISPR–Cas9-mediated genome editing
10.1038/s41587-024-02307-y · doi-reference
Utilizing AI-enhanced multi-omics integration for predictive modeling of disease susceptibility in functional phenotypes
10.53469/jtpes.2024.04(02).07 · doi-reference
10.2139/ssrn.6076076
10.2139/ssrn.6076076 · doi-reference
A comprehensive review of AAV-mediated strategies targeting microglia for therapeutic intervention of neurodegenerative diseases
10.1186/s12974-024-03232-2 · doi-reference
Nose to brain: exploring the progress of intranasal delivery of solid lipid nanoparticles and nanostructured lipid carriers
10.2147/ijn.s497480 · doi-reference
Hijacking of the nervous system in cancer: mechanism and therapeutic targets
10.1186/s12943-025-02246-5 · doi-reference
Applications of gold nanoparticles in brain diseases across the blood-brain barrier
10.2174/0929867329666220527121943 · doi-reference
TALEN-interceded genome editing in plants: unveiling new frontiers in secondary metabolite improvement and genetic diversity
10.3390/plants14193024 · doi-reference
Immune responses to adeno-associated virus vectors
10.2174/1566523054065039 · doi-reference
A review of mitochondrial dysfunction, pathophysiology and therapeutic prospects in neurodegenerative diseases
10.1186/s13064-025-00223-8 · doi-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 · doi-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 · doi-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 · doi-reference
Non-invasive CRISPR/Cas9 nanocapsules specifically edit α-synuclein for effective Parkinson’s disease treatment
10.1016/j.nantod.2025.102903 · doi-reference
Overcoming the blood–brain barrier: advanced strategies in targeted drug delivery for neurodegenerative diseases
10.3390/pharmaceutics17081041 · doi-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 · doi-reference
Hydrogels in gene delivery techniques for regenerative medicine and tissue engineering
10.1002/mabi.202300577 · doi-reference
Nucleic acid delivery by ionizable nanocarriers for brain disease treatment
10.1002/brx2.7 · doi-reference
Vectors in CRISPR gene editing for neurological disorders: challenges and opportunities
10.1002/adbi.202400374 · doi-reference
In vivo interactions of nucleic acid nanostructures with cells
10.1002/adma.202314232 · doi-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 · doi-reference
The blood–brain barrier cell-targeted gene delivery system to enhance nerve growth factor protein secretion in the brain
10.1021/acsbiomaterials.0c01113 · doi-reference
MRI-guided focused ultrasound for treating Parkinson’s disease with human mesenchymal stem cells
10.1038/s41598-025-85811-8 · doi-reference
Exosomes and biomimetic nanovesicles-mediated anti-glioblastoma therapy: a head-to-head comparison
10.1016/j.jconrel.2021.07.004 · doi-reference
Programmable gene insertion in human cells with a laboratory-evolved CRISPR-associated transposase
10.1126/science.adt5199 · doi-reference
Immunology of cell and gene therapy approaches for neurologic diseases
10.1016/b978-0-323-90120-8.00018-6 · doi-reference
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice
10.1016/j.ymthe.2025.05.010 · doi-reference
Treating Parkinson’s disease by astrocyte reprogramming: progress and challenges
10.1126/sciadv.abg3198 · doi-reference
Modifiers of GRN-associated frontotemporal lobar degeneration
10.1016/j.molmed.2017.08.004 · doi-reference
Recent advances and clinical prospects of non-viral brain-targeted gene delivery systems
10.1063/5.0255745 · doi-reference
Targeted delivery of hybrid nanovesicles for enhanced brain penetration to achieve synergistic therapy of glioma
10.1016/j.jconrel.2023.11.033 · doi-reference
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice
10.1038/s41586-022-04533-3 · doi-reference
Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?
10.1038/s41434-021-00232-2 · doi-reference
Mitochondrial-based therapies for neurodegenerative diseases: a review of the current literature
10.1007/s00210-025-04014-0 · doi-reference
Recombinant human myelin-associated glycoprotein promoter drives selective AAV-mediated transgene expression in oligodendrocytes
10.3389/fnmol.2016.00013 · doi-reference
In vitro CRISPR/Cas9-directed gene editing to model LRRK2 G2019S Parkinson’s disease in common marmosets
10.1038/s41598-020-60273-2 · doi-reference
Toward the development of epigenome editing-based therapeutics: potentials and challenges
10.3390/ijms24054778 · doi-reference