ArticleeLife2024
Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington's disease.
Abstract read
In one paragraph
Article in eLife, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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19citing papers in PubMed
–field-weighted citation impact
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
19 citing papers in PubMed.
- Base editing for precision therapeutics.Cell genomics · 2026Review
- Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- In vivo CRISPR base editing for treatment of Huntington's disease.Nature biomedical engineering · 2026Article
- Neuroprotective Effects of Transplanted Induced Pluripotent Stem Cell-Derived Neural Precursors in Huntington's Disease Models.International journal of stem cells · 2026Article
- Molecular features of a Huntington's disease knock-in minipig.Disease models & mechanisms · 2026Article
- In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression.Genome medicine · 2026Article
- Huntington disease: somatic expansion, pathobiology and therapeutics.Nature reviews. Neurology · 2026Review
- Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease.bioRxiv : the preprint server for biology · 2025Article
- Exploring immunotherapeutic strategies for neurodegenerative diseases: a focus on Huntington's disease and Prion diseases.Acta pharmacologica Sinica · 2025Review
- Base editing of trinucleotide repeats that cause Huntington's disease and Friedreich's ataxia reduces somatic repeat expansions in patient cells and in mice.Nature genetics · 2025Article
- Advances in gene and cellular therapeutic approaches for Huntington's disease.Protein & cell · 2025Review
- Tandem repeat disorders: from diagnosis to emerging therapeutic strategies.Encephalitis (Seoul, Korea) · 2025Article
- 3A or not 3A: Cytidine deaminases in the etiology of the CAG-repeat expansion diseases.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- APOBEC3A deaminates CTG hairpin loops to promote fragility and instability of expanded CAG/CTG repeats.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- CRISPR-Cas technologies in neurodegenerative disorders: mechanistic insights, therapeutic potential, and translational challenges.Frontiers in neurology · 2025Review
- Structural and Dynamical Properties of Nucleic Acid Hairpins Implicated in Trinucleotide Repeat Expansion Diseases.Biomolecules · 2024Review
- Therapeutic approaches targeting aging and cellular senescence in Huntington's disease.CNS neuroscience & therapeutics · 2024Review
- Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington's disease.eLife · 2024Article
- Precise editing of pathogenic nucleotide repeat expansions in iPSCs using paired prime editor.Nucleic acids research · 2024Article
Corrections and comments
- Update of
Authors and funding
10 authors.
Doo Eun ChoiCenter for Genomic Medicine, Massachusetts General Hospital, Boston, United States.
Jun Wan ShinCenter for Genomic Medicine, Massachusetts General Hospital, Boston, United States.
Sophia ZengCenter for Genomic Medicine, Massachusetts General Hospital, Boston, United States.
Eun Pyo HongCenter for Genomic Medicine, Massachusetts General Hospital, Boston, United States.ORCID https://orcid.org/0000-0001-7789-686X
Jae-Hyun JangCenter for Genomic Medicine, Massachusetts General Hospital, Boston, United States.
Jacob M LoupeCenter for Genomic Medicine, Massachusetts General Hospital, Boston, United States.
Vanessa C WheelerCenter for Genomic Medicine, Massachusetts General Hospital, Boston, United States.ORCID https://orcid.org/0000-0003-2619-589X
Hannah E StutzmanCenter for Genomic Medicine, Massachusetts General Hospital, Boston, United States.ORCID https://orcid.org/0000-0003-0500-437X
Ben KleinstiverCenter for Genomic Medicine, Massachusetts General Hospital, Boston, United States.
Jong-Min LeeCenter for Genomic Medicine, Massachusetts General Hospital, Boston, United States.ORCID https://orcid.org/0000-0001-5799-0787
Funding
Huntington's Disease Repeat Instability and PathogenesisR01NS049206 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI VANESSA C WHEELER · 2005 to 2026
$10.0MDisease-Modifying Genes in Huntington's DiseaseR01NS091161 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI JAMES F GUSELLA · 2015 to 2026
$7.2MTherapeutic Potential of Base Editing Strategies to Convert CAG to CAA in Huntington's DiseaseR01NS119471 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI LEE, JONG-MIN · 2021 to 2025
$2.6MGenetic foundation for complete mutant allele-specific CRISPR in neurodegenerative diseasesR01NS105709 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI LEE, JONG-MIN · 2018 to 2022
$2.4MNINDS NIH HHS NS049206NINDS NIH HHS NS105709NINDS NIH HHS NS119471NINDS NIH HHS R01 NS049206NINDS NIH HHS R01 NS105709NINDS NIH HHS R01 NS119471
Abstract
An expanded CAG repeat in the huntingtin gene (
Indexed as
Gene EditingHuntingtin ProteinHuntington DiseaseTrinucleotide Repeat ExpansionAnimalsDisease Models, AnimalGene Knock-In TechniquesHumansMiceMutationHtt protein, mouseHuntingtin Proteinbase editingCAG repeat instabilityCAG-to-CAA conversionduplicated interruptiongeneticsgenomicshumanHuntington's diseaseuninterrupted repeat
Identifiers
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Read underepoch 390
Registered trials
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.