Research graph
References from CRISPR‐based therapeutic and modelling approaches in Huntington's disease: Progress, challenges and future directions. Local targets link to admitted publications; unresolved targets remain external evidence.
Huntington's disease: a clinical review
10.1111/ene.13413 · 2018 · External reference
Clinical features of Huntington's disease
10.1007/978-3-319-71779-1_1 · 2018 · External reference
Overview of Huntington's disease and emerging treatment strategies: a narrative review
2025 · External reference
10.1007/978-3-031-73363-5_56
10.1007/978-3-031-73363-5_56 · 2025 · External reference
Potential disease‐modifying therapies for Huntington's disease: lessons learned and future opportunities
10.1016/s1474-4422(22)00121-1 · 2022 · External reference
Current and possible future therapeutic options for Huntington's disease
10.1177/11795735221092517 · 2022 · External reference
The potential of gene editing for Huntington's disease
10.1016/j.tins.2023.02.005 · 2023 · External reference
CRISPR/Cas9 mediated therapeutic approach in huntington's disease
10.1007/s12035-022-03150-5 · 2023 · External reference
CRISPR/Cas9 editing of the mutant huntingtin allele in vitro and in vivo
10.1016/j.ymthe.2016.11.010 · 2017 · External reference
Generation of new isogenic models of Huntington's disease using CRISPR‐Cas9 technology
10.3390/ijms21051854 · 2020 · External reference
Permanent inactivation of Huntington's disease mutation by personalized allele‐specific CRISPR/Cas9
2016 · External reference
Global huntingtin knockout in adult mice leads to fatal neurodegeneration that spares the pancreas
10.26508/lsa.202402571 · 2024 · External reference
Long somatic DNA‐repeat expansion drives neurodegeneration in Huntington's disease
10.1016/j.cell.2024.11.038 · 2025 · External reference
CRISPR‐based genome‐editing tools for Huntington's disease research and therapy
10.1007/s12264-022-00880-3 · 2022 · External reference
Past, present, and future of CRISPR genome editing technologies
10.1016/j.cell.2024.01.042 · 2024 · External reference
CRISPR interference efficiently induces specific and reversible gene silencing in human iPSCs
10.1016/j.stem.2016.01.022 · 2016 · External reference
DNA double‐strand break‐free CRISPR interference delays Huntington's disease progression in mice
10.1038/s42003-023-04829-8 · 2023 · External reference
CRISPR interference (CRISPRi) for sequence‐specific control of gene expression
10.1038/nprot.2013.132 · 2013 · External reference
Transcriptome engineering with RNA‐targeting type VI‐D CRISPR effectors
10.1016/j.cell.2018.02.033 · 2018 · 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
CRISPR‐Cas12a induced DNA double‐strand breaks are repaired by multiple pathways with different mutation profiles in Magnaporthe oryzae
10.1038/s41467-022-34736-1 · 2022 · External reference
Building CRISPR gene therapies for the central nervous system: a review
10.1001/jamaneurol.2023.4983 · 2024 · External reference
CRISPR/Cas9 landscape: current state and future perspectives
10.3390/ijms242216077 · 2023 · External reference
CRISPR‐based therapeutic genome editing: strategies and in vivo delivery by AAV vectors
10.1016/j.cell.2020.03.023 · 2020 · External reference
An insight into allele‐selective approaches to lowering mutant huntingtin protein for Huntington's disease treatment
10.1016/j.biopha.2024.117557 · 2024 · External reference
CRISPR/Cas9‐mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease
10.1172/jci92087 · 2017 · External reference
CRISPR‐Cas9‐mediated genome editing increases lifespan and improves motor deficits in a Huntington's disease mouse model
10.1016/j.omtn.2019.07.009 · 2019 · External reference
Cpf1 is a single RNA‐guided endonuclease of a class 2 CRISPR‐Cas system
10.1016/j.cell.2015.09.038 · 2015 · External reference
The biology of huntingtin
10.1016/j.neuron.2016.02.003 · 2016 · External reference
Huntingtin and the synapse
10.3389/fncel.2021.689332 · 2021 · External reference
Gene targeting techniques for Huntington's disease
10.1016/j.arr.2021.101385 · 2021 · External reference
PAM‐altering SNP‐based allele‐specific CRISPR‐Cas9 therapeutic strategies for Huntington's disease
10.1016/j.omtm.2022.08.005 · 2022 · External reference
Huntingtin exon 1 deletion does not alter the subcellular distribution of huntingtin and gene transcription in mice
10.3389/fncel.2022.1021592 · 2022 · External reference
10.64898/2026.08.10.744034
10.64898/2026.08.10.744034 · External reference
Mutation‐independent allele‐specific editing by CRISPR‐Cas9, a novel approach to treat autosomal dominant disease
10.1016/j.ymthe.2020.05.002 · 2020 · External reference
Huntingtin‐lowering therapies for huntington disease: a review of the evidence of potential benefits and risks
10.1001/jamaneurol.2020.0299 · 2020 · External reference
Single‐base resolution: increasing the specificity of the CRISPR‐Cas system in gene editing
10.1016/j.ymthe.2020.11.009 · 2021 · External reference
Limitations of dual‐single guide RNA CRISPR strategies for the treatment of central nervous system genetic disorders
10.1089/hum.2023.109 · 2023 · External reference
DNA targeting specificity of RNA‐guided Cas9 nucleases
10.1038/nbt.2647 · 2013 · External reference
C2c2 is a single‐component programmable RNA‐guided RNA‐targeting CRISPR effector
10.1126/science.aaf5573 · 2016 · External reference
RNA editing with CRISPR‐Cas13
10.1126/science.aaq0180 · 2017 · External reference
RNA‐targeting CRISPR/CasRx system relieves disease symptoms in Huntington's disease models
10.1186/s13024-024-00794-w · 2025 · 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
Chemically modified guide RNAs enhance CRISPR‐Cas13 knockdown in human cells
10.1016/j.chembiol.2021.07.011 · 2022 · External reference
Precise RNA targeting with CRISPR‐Cas13d
10.1038/s41587-025-02558-3 · 2026 · External reference
Negative autoregulation mitigates collateral RNase activity of repeat‐targeting CRISPR‐Cas13d in mammalian cells
10.1016/j.celrep.2022.111226 · 2022 · External reference
CRISPR‐Cas13: pioneering RNA editing for nucleic acid therapeutics
10.34133/bdr.0041 · 2024 · External reference
Molecular mechanisms of RNA targeting by Cas13‐containing type VI CRISPR‐Cas systems
10.1016/j.jmb.2018.06.029 · 2019 · External reference
Cas13d is a compact RNA‐targeting type VI CRISPR effector positively modulated by a WYL‐domain‐containing accessory protein
10.1016/j.molcel.2018.02.028 · 2018 · External reference
Huntington's disease mouse models: unraveling the pathology caused by CAG repeat expansion
10.12703/r/10-77 · 2021 · External reference
An enhanced Q175 knock‐in mouse model of Huntington disease with higher mutant huntingtin levels and accelerated disease phenotypes
10.1093/hmg/ddw212 · 2016 · External reference
Huntingtin silencing delays onset and slows progression of Huntington's disease: a biomarker study
10.1093/brain/awab190 · 2021 · External reference
Unbiased profiling of isogenic Huntington disease hPSC‐derived CNS and peripheral cells reveals strong cell‐type specificity of CAG length effects
10.1016/j.celrep.2019.02.008 · 2019 · External reference
A Huntingtin knockin pig model recapitulates features of selective neurodegeneration in Huntington's disease
10.1016/j.cell.2018.03.005 · 2018 · External reference
Cas9‐mediated replacement of expanded CAG repeats in a pig model of Huntington's disease
10.1038/s41551-023-01007-3 · 2023 · External reference
Generation of rhesus macaque embryos with expanded CAG trinucleotide repeats in the huntingtin gene
10.3390/cells13100829 · 2024 · External reference
Reversal of phenotypic abnormalities by CRISPR/Cas9‐mediated gene correction in Huntington disease patient‐derived induced pluripotent stem cells
10.1016/j.stemcr.2017.01.022 · 2017 · External reference
Patient‐specific iPSC‐based models of Huntington's disease as a tool to study store‐operated calcium entry drug targeting
10.3389/fphar.2018.00696 · 2018 · External reference
Huntington's disease cellular phenotypes are rescued non‐cell autonomously by healthy cells in mosaic telencephalic organoids
10.1038/s41467-024-50877-x · 2024 · External reference
Large animal models of Huntington's disease: what we have learned and where we need to go next
10.3233/jhd-200425 · 2020 · External reference
Genetic modifiers of somatic expansion and clinical phenotypes in Huntington's disease highlight shared and tissue‐specific effects
10.1038/s41588-025-02191-5 · 2025 · External reference
CAG repeat not polyglutamine length determines timing of Huntington's disease onset
10.1016/j.cell.2019.06.036 · 2019 · External reference
In vivo CRISPR‐Cas9 genome editing in mice identifies genetic modifiers of somatic CAG repeat instability in Huntington's disease
10.1038/s41588-024-02054-5 · 2025 · External reference
FAN1 modifies Huntington's disease progression by stabilizing the expanded HTT CAG repeat
10.1093/hmg/ddy375 · 2019 · External reference
Di‐valent siRNA‐mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease
10.1016/j.ymthe.2023.05.006 · 2023 · 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
Huntington disease: somatic expansion, pathobiology and therapeutics
10.1038/s41582-025-01159-7 · 2026 · External reference
Therapeutic validation of MMR‐associated genetic modifiers in a human ex vivo model of Huntington disease
10.1016/j.ajhg.2024.04.015 · 2024 · External reference
Base editing strategies to convert CAG to CAA diminish the disease‐causing mutation in Huntington's disease
10.7554/elife.89782.2 · 2024 · External reference
Base editing of trinucleotide repeats that cause Huntington's disease and Friedreich's ataxia reduces somatic repeat expansions in patient cells and in mice
10.1038/s41588-025-02172-8 · 2025 · External reference
Self‐inactivating AAV‐CRISPR at different ages enables sustained amelioration of Huntington's disease deficits in BAC226Q mice
10.1126/sciadv.aea8052 · 2026 · External reference
Treatment of Huntington's disease with a pan‐HTT‐targeting CRISPR nuclease
10.1016/j.ymthe.2026.04.038 · 2026 · External reference
Lipid nanoparticles enhance mRNA delivery to the central nervous system upon intrathecal injection
10.1002/adma.202417097 · 2025 · External reference
Therapeutic in vivo genome editing: innovations and challenges in rAAV vector‐based CRISPR delivery
10.1038/s41434-025-00573-2 · 2026 · External reference
Immunogenicity and toxicity of AAV gene therapy
10.3389/fimmu.2022.975803 · 2022 · External reference
AAV engineering for improving tropism to the central nervous system
2023 · External reference
Designing and optimizing AAV‐mediated gene therapy for neurodegenerative diseases: from bench to bedside
10.1186/s12967-024-05661-2 · 2024 · External reference
Recent progress and considerations for AAV gene therapies targeting the central nervous system
10.1186/s11689-018-9234-0 · 2018 · External reference
Circumventing the packaging limit of AAV‐mediated gene replacement therapy for neurological disorders
10.1080/14712598.2022.2012148 · 2022 · External reference
Adeno‐associated virus (AAV) dual vector strategies for gene therapy encoding large transgenes
2017 · External reference
Engineering adeno‐associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing
10.1016/j.biomaterials.2025.123314 · 2025 · External reference
A novel class of self‐complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR
10.1016/j.omtm.2024.101206 · 2024 · External reference
Immune toxicities in AAV gene therapy: overview for clinicians
10.3390/ijms27073196 · 2026 · 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
Lung and liver editing by lipid nanoparticle delivery of a stable CRISPR‐Cas9 ribonucleoprotein
10.1038/s41587-024-02437-3 · 2025 · External reference
Comparative analysis of lipid nanoparticle‐mediated delivery of CRISPR‐Cas9 RNP versus mRNA/sgRNA for gene editing in vitro and in vivo
10.1016/j.ejpb.2024.114207 · 2024 · External reference
CRISPR‐Cas9 in vivo gene editing for transthyretin amyloidosis
10.1056/nejmoa2107454 · 2021 · External reference
Highly efficient RNA‐guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins
10.1101/gr.171322.113 · 2014 · External reference
Drug delivery systems for CRISPR‐based genome editors
10.1038/s41573-023-00762-x · 2023 · External reference
Lipid nanoparticles for mRNA delivery
10.1038/s41578-021-00358-0 · 2021 · External reference
Immunogenicity of lipid nanoparticles and its impact on the efficacy of mRNA vaccines and therapeutics
10.1038/s12276-023-01086-x · 2023 · 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
Lipid nanoparticles deliver mRNA to the blood–brain barrier
10.1007/s12274-024-6827-7 · 2024 · External reference
Blood‐brain‐barrier‐crossing lipid nanoparticles for mRNA delivery to the central nervous system
10.1038/s41563-024-02114-5 · 2025 · External reference
Lipid nanoparticles for mRNA delivery in brain via systemic administration
10.1126/sciadv.adw0730 · 2025 · External reference
Focused ultrasound‐mediated brain genome editing
10.1073/pnas.2302910120 · 2023 · External reference
Customizable virus‐like particles deliver CRISPR–Cas9 ribonucleoprotein for effective ocular neovascular and Huntington's disease gene therapy
10.1038/s41565-024-01851-7 · 2025 · External reference
Engineered virus‐like particles for efficient in vivo delivery of therapeutic proteins
10.1016/j.cell.2021.12.021 · 2022 · External reference
Repair of double‐strand breaks induced by CRISPR‐Cas9 leads to large deletions and complex rearrangements
10.1038/nbt.4192 · 2018 · External reference
Unintended CRISPR‐Cas9 editing outcomes: a review of the detection and prevalence of structural variants generated by gene‐editing in human cells
10.1007/s00439-023-02561-1 · 2023 · External reference
Chromothripsis as an on‐target consequence of CRISPR–Cas9 genome editing
10.1038/s41588-021-00838-7 · 2021 · External reference
Collateral activity of the CRISPR/RfxCas13d system in human cells
10.1038/s42003-023-04708-2 · 2023 · External reference
High‐fidelity Cas13 variants for targeted RNA degradation with minimal collateral effects
10.1038/s41587-022-01419-7 · 2023 · External reference
Identification of preexisting adaptive immunity to Cas9 proteins in humans
10.1038/s41591-018-0326-x · 2019 · External reference
High prevalence of Streptococcus pyogenes Cas9‐reactive T cells within the adult human population
10.1038/s41591-018-0204-6 · 2019 · External reference
Pre‐existing adaptive immunity to the RNA‐editing enzyme Cas13d in humans
10.1038/s41591-022-01848-6 · 2022 · External reference
DNA mismatch repair and its role in Huntington's disease
10.3233/jhd-200438 · 2021 · External reference
Somatic CAG repeat expansion in blood associates with biomarkers of neurodegeneration in Huntington's disease decades before clinical motor diagnosis
10.1038/s41591-024-03424-6 · 2025 · External reference
Heritable human genome editing: research progress, ethical considerations, and hurdles to clinical practice
10.1016/j.cell.2021.02.036 · 2021 · External reference
Setting ethical limits on human gene editing after the fall of the somatic/germline barrier
10.1073/pnas.2004837117 · 2021 · External reference
40+ years of the Huntington disease predictive genetic testing protocol: recommendations for an update
10.1002/jgc4.70226 · 2026 · External reference
Family communication patterns and challenges of Huntington's disease risk, the decision to pursue presymptomatic testing, and test results
10.3233/jhd-200402 · 2020 · External reference
Haplotyping SNPs for allele‐specific gene editing of the expanded huntingtin allele using long‐read sequencing
2023 · External reference
A biological classification of Huntington's disease: the integrated staging system
10.1016/s1474-4422(22)00120-x · 2022 · External reference
Evaluation of mutant huntingtin and neurofilament proteins as potential markers in Huntington's disease
10.1126/scitranslmed.aat7108 · 2018 · External reference
Mutant huntingtin and neurofilament light have distinct longitudinal dynamics in Huntington's disease
10.1126/scitranslmed.abc2888 · 2020 · External reference
Clinical pharmacology and translational considerations in the development of CRISPR‐based therapies
10.1002/cpt.3000 · 2023 · External reference
Regulatory considerations for clinical trial applications with CRISPR‐based medicinal products
10.1089/crispr.2021.0148 · 2022 · External reference
Chronic in vivo CRISPR‐Cas genome editing: challenges, long‐term safety, and outlook
10.3390/cells15020156 · 2026 · External reference
Huntingtin‐lowering therapies for huntington disease: a review of the evidence of potential benefits and risks
10.1001/jamaneurol.2020.0299 · ExternalCitation · doi-reference
Building CRISPR gene therapies for the central nervous system: a review
10.1001/jamaneurol.2023.4983 · ExternalCitation · doi-reference
Lipid nanoparticles enhance mRNA delivery to the central nervous system upon intrathecal injection
10.1002/adma.202417097 · ExternalCitation · doi-reference
Clinical pharmacology and translational considerations in the development of CRISPR‐based therapies
10.1002/cpt.3000 · ExternalCitation · doi-reference
40+ years of the Huntington disease predictive genetic testing protocol: recommendations for an update
10.1002/jgc4.70226 · ExternalCitation · doi-reference
10.1007/978-3-031-73363-5_56
10.1007/978-3-031-73363-5_56 · ExternalCitation · doi-reference
Clinical features of Huntington's disease
10.1007/978-3-319-71779-1_1 · ExternalCitation · doi-reference
Unintended CRISPR‐Cas9 editing outcomes: a review of the detection and prevalence of structural variants generated by gene‐editing in human cells
10.1007/s00439-023-02561-1 · ExternalCitation · doi-reference
CRISPR/Cas9 mediated therapeutic approach in huntington's disease
10.1007/s12035-022-03150-5 · ExternalCitation · doi-reference
CRISPR‐based genome‐editing tools for Huntington's disease research and therapy
10.1007/s12264-022-00880-3 · ExternalCitation · doi-reference
Lipid nanoparticles deliver mRNA to the blood–brain barrier
10.1007/s12274-024-6827-7 · ExternalCitation · doi-reference
Therapeutic validation of MMR‐associated genetic modifiers in a human ex vivo model of Huntington disease
10.1016/j.ajhg.2024.04.015 · ExternalCitation · doi-reference
Gene targeting techniques for Huntington's disease
10.1016/j.arr.2021.101385 · ExternalCitation · doi-reference
Engineering adeno‐associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing
10.1016/j.biomaterials.2025.123314 · ExternalCitation · doi-reference
An insight into allele‐selective approaches to lowering mutant huntingtin protein for Huntington's disease treatment
10.1016/j.biopha.2024.117557 · ExternalCitation · doi-reference
Cpf1 is a single RNA‐guided endonuclease of a class 2 CRISPR‐Cas system
10.1016/j.cell.2015.09.038 · ExternalCitation · doi-reference
Transcriptome engineering with RNA‐targeting type VI‐D CRISPR effectors
10.1016/j.cell.2018.02.033 · ExternalCitation · doi-reference
A Huntingtin knockin pig model recapitulates features of selective neurodegeneration in Huntington's disease
10.1016/j.cell.2018.03.005 · ExternalCitation · doi-reference
CAG repeat not polyglutamine length determines timing of Huntington's disease onset
10.1016/j.cell.2019.06.036 · ExternalCitation · doi-reference
CRISPR‐based therapeutic genome editing: strategies and in vivo delivery by AAV vectors
10.1016/j.cell.2020.03.023 · ExternalCitation · doi-reference
Heritable human genome editing: research progress, ethical considerations, and hurdles to clinical practice
10.1016/j.cell.2021.02.036 · ExternalCitation · doi-reference
Engineered virus‐like particles for efficient in vivo delivery of therapeutic proteins
10.1016/j.cell.2021.12.021 · ExternalCitation · doi-reference
Past, present, and future of CRISPR genome editing technologies
10.1016/j.cell.2024.01.042 · ExternalCitation · doi-reference
Long somatic DNA‐repeat expansion drives neurodegeneration in Huntington's disease
10.1016/j.cell.2024.11.038 · ExternalCitation · doi-reference
Unbiased profiling of isogenic Huntington disease hPSC‐derived CNS and peripheral cells reveals strong cell‐type specificity of CAG length effects
10.1016/j.celrep.2019.02.008 · ExternalCitation · doi-reference
Negative autoregulation mitigates collateral RNase activity of repeat‐targeting CRISPR‐Cas13d in mammalian cells
10.1016/j.celrep.2022.111226 · ExternalCitation · doi-reference
Chemically modified guide RNAs enhance CRISPR‐Cas13 knockdown in human cells
10.1016/j.chembiol.2021.07.011 · ExternalCitation · doi-reference
Comparative analysis of lipid nanoparticle‐mediated delivery of CRISPR‐Cas9 RNP versus mRNA/sgRNA for gene editing in vitro and in vivo
10.1016/j.ejpb.2024.114207 · ExternalCitation · doi-reference
Molecular mechanisms of RNA targeting by Cas13‐containing type VI CRISPR‐Cas systems
10.1016/j.jmb.2018.06.029 · ExternalCitation · doi-reference
Cas13d is a compact RNA‐targeting type VI CRISPR effector positively modulated by a WYL‐domain‐containing accessory protein
10.1016/j.molcel.2018.02.028 · ExternalCitation · doi-reference
The biology of huntingtin
10.1016/j.neuron.2016.02.003 · ExternalCitation · doi-reference
PAM‐altering SNP‐based allele‐specific CRISPR‐Cas9 therapeutic strategies for Huntington's disease
10.1016/j.omtm.2022.08.005 · ExternalCitation · doi-reference
A novel class of self‐complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR
10.1016/j.omtm.2024.101206 · ExternalCitation · doi-reference
CRISPR‐Cas9‐mediated genome editing increases lifespan and improves motor deficits in a Huntington's disease mouse model
10.1016/j.omtn.2019.07.009 · ExternalCitation · doi-reference
CRISPR interference efficiently induces specific and reversible gene silencing in human iPSCs
10.1016/j.stem.2016.01.022 · ExternalCitation · doi-reference
Reversal of phenotypic abnormalities by CRISPR/Cas9‐mediated gene correction in Huntington disease patient‐derived induced pluripotent stem cells
10.1016/j.stemcr.2017.01.022 · ExternalCitation · doi-reference
The potential of gene editing for Huntington's disease
10.1016/j.tins.2023.02.005 · ExternalCitation · doi-reference
CRISPR/Cas9 editing of the mutant huntingtin allele in vitro and in vivo
10.1016/j.ymthe.2016.11.010 · ExternalCitation · doi-reference
Mutation‐independent allele‐specific editing by CRISPR‐Cas9, a novel approach to treat autosomal dominant disease
10.1016/j.ymthe.2020.05.002 · ExternalCitation · doi-reference
Single‐base resolution: increasing the specificity of the CRISPR‐Cas system in gene editing
10.1016/j.ymthe.2020.11.009 · ExternalCitation · doi-reference
Di‐valent siRNA‐mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease
10.1016/j.ymthe.2023.05.006 · ExternalCitation · doi-reference
Treatment of Huntington's disease with a pan‐HTT‐targeting CRISPR nuclease
10.1016/j.ymthe.2026.04.038 · ExternalCitation · doi-reference
A biological classification of Huntington's disease: the integrated staging system
10.1016/s1474-4422(22)00120-x · ExternalCitation · doi-reference
Potential disease‐modifying therapies for Huntington's disease: lessons learned and future opportunities
10.1016/s1474-4422(22)00121-1 · ExternalCitation · doi-reference
Brain nucleic acid delivery and genome editing via focused ultrasound‐mediated blood–brain barrier opening and long‐circulating nanoparticles
10.1021/acsnano.4c05270 · ExternalCitation · doi-reference
DNA targeting specificity of RNA‐guided Cas9 nucleases
10.1038/nbt.2647 · ExternalCitation · doi-reference
Repair of double‐strand breaks induced by CRISPR‐Cas9 leads to large deletions and complex rearrangements
10.1038/nbt.4192 · ExternalCitation · doi-reference
CRISPR interference (CRISPRi) for sequence‐specific control of gene expression
10.1038/nprot.2013.132 · ExternalCitation · doi-reference
Immunogenicity of lipid nanoparticles and its impact on the efficacy of mRNA vaccines and therapeutics
10.1038/s12276-023-01086-x · ExternalCitation · doi-reference
Therapeutic in vivo genome editing: innovations and challenges in rAAV vector‐based CRISPR delivery
10.1038/s41434-025-00573-2 · ExternalCitation · doi-reference
CRISPR‐Cas12a induced DNA double‐strand breaks are repaired by multiple pathways with different mutation profiles in Magnaporthe oryzae
10.1038/s41467-022-34736-1 · ExternalCitation · doi-reference
Huntington's disease cellular phenotypes are rescued non‐cell autonomously by healthy cells in mosaic telencephalic organoids
10.1038/s41467-024-50877-x · ExternalCitation · doi-reference
Cas9‐mediated replacement of expanded CAG repeats in a pig model of Huntington's disease
10.1038/s41551-023-01007-3 · ExternalCitation · doi-reference
Blood‐brain‐barrier‐crossing lipid nanoparticles for mRNA delivery to the central nervous system
10.1038/s41563-024-02114-5 · ExternalCitation · doi-reference
Customizable virus‐like particles deliver CRISPR–Cas9 ribonucleoprotein for effective ocular neovascular and Huntington's disease gene therapy
10.1038/s41565-024-01851-7 · ExternalCitation · doi-reference
Drug delivery systems for CRISPR‐based genome editors
10.1038/s41573-023-00762-x · ExternalCitation · doi-reference
Lipid nanoparticles for mRNA delivery
10.1038/s41578-021-00358-0 · ExternalCitation · doi-reference
Huntington disease: somatic expansion, pathobiology and therapeutics
10.1038/s41582-025-01159-7 · ExternalCitation · doi-reference
High‐fidelity Cas13 variants for targeted RNA degradation with minimal collateral effects
10.1038/s41587-022-01419-7 · ExternalCitation · doi-reference
Lung and liver editing by lipid nanoparticle delivery of a stable CRISPR‐Cas9 ribonucleoprotein
10.1038/s41587-024-02437-3 · ExternalCitation · doi-reference
Precise RNA targeting with CRISPR‐Cas13d
10.1038/s41587-025-02558-3 · ExternalCitation · doi-reference
Chromothripsis as an on‐target consequence of CRISPR–Cas9 genome editing
10.1038/s41588-021-00838-7 · ExternalCitation · doi-reference
In vivo CRISPR‐Cas9 genome editing in mice identifies genetic modifiers of somatic CAG repeat instability in Huntington's disease
10.1038/s41588-024-02054-5 · ExternalCitation · doi-reference
Base editing of trinucleotide repeats that cause Huntington's disease and Friedreich's ataxia reduces somatic repeat expansions in patient cells and in mice
10.1038/s41588-025-02172-8 · ExternalCitation · doi-reference
Genetic modifiers of somatic expansion and clinical phenotypes in Huntington's disease highlight shared and tissue‐specific effects
10.1038/s41588-025-02191-5 · ExternalCitation · doi-reference
High prevalence of Streptococcus pyogenes Cas9‐reactive T cells within the adult human population
10.1038/s41591-018-0204-6 · ExternalCitation · doi-reference
Identification of preexisting adaptive immunity to Cas9 proteins in humans
10.1038/s41591-018-0326-x · ExternalCitation · doi-reference
Pre‐existing adaptive immunity to the RNA‐editing enzyme Cas13d in humans
10.1038/s41591-022-01848-6 · ExternalCitation · doi-reference
Somatic CAG repeat expansion in blood associates with biomarkers of neurodegeneration in Huntington's disease decades before clinical motor diagnosis
10.1038/s41591-024-03424-6 · ExternalCitation · doi-reference
An RNA‐targeting CRISPR‐Cas13d system alleviates disease‐related phenotypes in Huntington's disease models
10.1038/s41593-022-01207-1 · ExternalCitation · doi-reference
Collateral activity of the CRISPR/RfxCas13d system in human cells
10.1038/s42003-023-04708-2 · ExternalCitation · doi-reference
DNA double‐strand break‐free CRISPR interference delays Huntington's disease progression in mice
10.1038/s42003-023-04829-8 · ExternalCitation · doi-reference
CRISPR‐Cas9 in vivo gene editing for transthyretin amyloidosis
10.1056/nejmoa2107454 · ExternalCitation · doi-reference
Setting ethical limits on human gene editing after the fall of the somatic/germline barrier
10.1073/pnas.2004837117 · ExternalCitation · doi-reference
Focused ultrasound‐mediated brain genome editing
10.1073/pnas.2302910120 · ExternalCitation · doi-reference
Circumventing the packaging limit of AAV‐mediated gene replacement therapy for neurological disorders
10.1080/14712598.2022.2012148 · ExternalCitation · doi-reference
Regulatory considerations for clinical trial applications with CRISPR‐based medicinal products
10.1089/crispr.2021.0148 · ExternalCitation · doi-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 · ExternalCitation · doi-reference
Limitations of dual‐single guide RNA CRISPR strategies for the treatment of central nervous system genetic disorders
10.1089/hum.2023.109 · ExternalCitation · doi-reference
Huntingtin silencing delays onset and slows progression of Huntington's disease: a biomarker study
10.1093/brain/awab190 · ExternalCitation · doi-reference
Exon 1‐targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington's disease models
10.1093/brain/awae266 · ExternalCitation · doi-reference
An enhanced Q175 knock‐in mouse model of Huntington disease with higher mutant huntingtin levels and accelerated disease phenotypes
10.1093/hmg/ddw212 · ExternalCitation · doi-reference
FAN1 modifies Huntington's disease progression by stabilizing the expanded HTT CAG repeat
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