ArticleNature communications2025
A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTD.
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Article in Nature communications, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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14citing papers in PubMed
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Who cites it
14 citing papers in PubMed.
- Insights into X-linked Dystonia-Parkinsonism and Spinocerebellar Ataxia type 36 Through the Lens of Amyotrophic Lateral Sclerosis/Frontotemporal Dementia.Molecular neurobiology · 2026Review
- RNA therapeutics: current status and future directions.Signal transduction and targeted therapy · 2026Review
- Adenovirus vector-mediated delivery of the CRISPR-CasRx system enables gene knockdown with suppressed collateral activity.Molecular therapy. Nucleic acids · 2026Article
- Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Gene Targeted Therapies for Neurodegenerative Disorders: Strategies and Implications in ALS and SMA.Genes · 2026Review
- Precise RNA targeting with CRISPR-Cas13d.Nature biotechnology · 2026Article
- Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease.bioRxiv : the preprint server for biology · 2025Article
- Nanomedicine-enhanced delivery of CRISPR-Cas13 for RNA editing in C9orf72-associated ALS.Annals of medicine and surgery (2012) · 2025Article
- CRISPR in Neurodegenerative Diseases Treatment: An Alternative Approach to Current Therapies.Genes · 2025Review
- Review
- RNA-Targeting CRISPR/CasRx system relieves disease symptoms in Huntington's disease models.Molecular neurodegeneration · 2025Article
- A Manual for Genome and Transcriptome Engineering.IEEE reviews in biomedical engineering · 2025Review
- Article
- PURA syndrome-a genetic cause of a neurodevelopmental disorder-case report.Frontiers in pediatrics · 2025Article
Corrections and comments
- Update of
Authors and funding
12 authors.
Tristan X McCallisterDepartment of Bioengineering, The Grainger College of Engineering, University of Illinois Urbana-Champaign, Urbana, IL, USA.
Colin K W LimDepartment of Bioengineering, The Grainger College of Engineering, University of Illinois Urbana-Champaign, Urbana, IL, USA.
Mayuri SinghDepartment of Bioengineering, The Grainger College of Engineering, University of Illinois Urbana-Champaign, Urbana, IL, USA.
Sijia ZhangDepartment of Bioengineering, The Grainger College of Engineering, University of Illinois Urbana-Champaign, Urbana, IL, USA.
Najah S AhsanDepartment of Bioengineering, The Grainger College of Engineering, University of Illinois Urbana-Champaign, Urbana, IL, USA.
William M TerpstraDepartment of Bioengineering, The Grainger College of Engineering, University of Illinois Urbana-Champaign, Urbana, IL, USA.
Alisha Y XiongDepartment of Bioengineering, The Grainger College of Engineering, University of Illinois Urbana-Champaign, Urbana, IL, USA.ORCID http://orcid.org/0009-0000-3269-3081
M Alejandra Zeballos CDepartment of Bioengineering, The Grainger College of Engineering, University of Illinois Urbana-Champaign, Urbana, IL, USA.ORCID http://orcid.org/0000-0002-5194-2909
Jackson E PowellDepartment of Bioengineering, The Grainger College of Engineering, University of Illinois Urbana-Champaign, Urbana, IL, USA.
Jenny DrnevichHigh-Performance Biological Computing, Roy J. Carver Biotechnology Center, University of Illinois Urbana-Champaign, Urbana, IL, USA.ORCID http://orcid.org/0000-0002-1401-8311
Yifei KangHigh-Performance Biological Computing, Roy J. Carver Biotechnology Center, University of Illinois Urbana-Champaign, Urbana, IL, USA.
Thomas GajDepartment of Bioengineering, The Grainger College of Engineering, University of Illinois Urbana-Champaign, Urbana, IL, USA. gaj@illinois.edu.ORCID http://orcid.org/0000-0001-6004-9664
Funding
Optimization of an in vivo base editing strategy to treat SOD1-linked ALSU01NS122102 · NINDS · UNIVERSITY OF ILLINOIS AT URBANA-CHAMPAIGN · PI Thomas Gaj, Pablo Perez-Pinera · 2022 to 2026
$5.0MDevelopment of Technologies for Efficient In Vivo Prime EditingR01GM141296 · NIGMS · UNIVERSITY OF ILLINOIS AT URBANA-CHAMPAIGN · PI PEREZ-PINERA, PABLO, SONG, JUN S · 2021 to 2024
$2.1MTissue microenvironment (TIMe) training programT32EB019944 · NIBIB · UNIVERSITY OF ILLINOIS AT URBANA-CHAMPAIGN · PI BHARGAVA, ROHIT, GASKINS, REX · 2016 to 2025
$1.9MDevelopment of a CRISPR-Cas13 Gene Therapy for SOD1-Linked ALSR01NS123556 · NINDS · UNIVERSITY OF ILLINOIS AT URBANA-CHAMPAIGN · PI Thomas Gaj · 2022 to 2026
$1.8MAmyotrophic Lateral Sclerosis Association (ALS Association) 20-IIP-516Muscular Dystrophy Association (Muscular Dystrophy Association Inc.) MDA602798NIBIB NIH HHS T32 EB019944NIGMS NIH HHS R01 GM141296NINDS NIH HHS R01 NS123556NINDS NIH HHS U01 NS122102U.S. Department of Health & Human Services | NIH | National Institute of General Medical Sciences (NIGMS) 5R01GM141296U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (NINDS) 1R01NS123556-01A1U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (NINDS) 1U01NS122102-01A1
Abstract
An abnormal expansion of a GGGGCC (G
Indexed as
Amyotrophic Lateral SclerosisC9orf72 ProteinCRISPR-Cas SystemsFrontotemporal DementiaAnimalsDisease Models, AnimalDNA Repeat ExpansionHumansMiceMotor NeuronsC9orf72 ProteinC9orf72 protein, human
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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.