ArticleScience (New York, N.Y.)2023
Base editing rescue of spinal muscular atrophy in cells and in mice.
Open access · hybridAbstract read
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Article in Science (New York, N.Y.), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 84 papers.
0numbers the graph read from it
0cells of the map it votes in
84citing papers in PubMed
35.9field-weighted citation impact, top 1% of its field
What it found
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Who cites it
84 citing papers in PubMed, 125 citations in OpenAlex.
- Base editing rescues a hereditary motor neuron disease in mouse and patient-derived iPSC organoid models.Molecular therapy. Advances · 2026Article
- Base editing for precision therapeutics.Cell genomics · 2026Review
- Mechanistic machine learning for prediction of prime editing outcomes.Nature biotechnology · 2026Article
- UNCOVERseq enables sensitive and controlled gene editing off-target nomination across CRISPR-Cas modalities and systems.Nature communications · 2026Article
- Incorporating AI-optimized zinc finger proteins enhances the efficiencies and targeting ranges of miniature base editors.Nature communications · 2026Article
- Therapeutic adenine base editor with minimized off-target effects.Protein & cell · 2026Article
- Long-term comparative analysis of AAV9-mediated gene replacement therapies for spinal muscular atrophy in mice.Nature communications · 2026Article
- Spatial profiling of gene editing by in situ sequencing in mice and macaques.Nature biomedical engineering · 2026Article
- In vivo base editing rescues liver pathophysiology and peroxisome dysfunction in a mouse model of Zellweger spectrum disorder.Nature biomedical engineering · 2026Article
- Haloperidol induces neuroprotection and enhances neuromuscular function in both murine and human models of spinal muscular atrophy.Experimental & molecular medicine · 2026Article
- Gene Targeted Therapies for Neurodegenerative Disorders: Strategies and Implications in ALS and SMA.Genes · 2026Review
- Article
- Mechanistic machine learning enables interpretable and generalizable prediction of prime editing outcomes.bioRxiv : the preprint server for biology · 2026Article
- A hit for base editing: treatment of developmental epilepsy in a mouse model.The Journal of clinical investigation · 2026Article
- Base editing rescues seizures and sudden death in a SCN8A mutation-associated developmental epileptic encephalopathy model.The Journal of clinical investigation · 2026Article
- RNA editing for the treatment of alpha-1 antitrypsin deficiency.Nucleic acids research · 2026Article
- Sensitive and unbiased genome-wide profiling of base-editor-induced off-target activity using CHANGE-seq-BE.Nature biotechnology · 2026Article
- Review
- Genetic therapies for neurological diseases.Pharmacological reviews · 2026Review
- Spinal muscular atrophy in the disease-modifying therapy era: successes, limitations and future directions.Frontiers in molecular medicine · 2026Review
24 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
Authors and funding
17 authors at 4 institutions in 1 country.
Mandana Arbab *Department of Neurology, Rosamund Stone Zander Translational Neuroscience Center, Boston Children's Hospital, Boston, MA 02115, USA.ORCID 0000-0001-9216-5096
Zaneta Matuszek *Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0001-5627-0593
Kaitlyn M KrayDepartment of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.ORCID 0000-0002-6025-3128
Ailing DuHorae Gene Therapy Center, UMass Chan Medical School, University of Massachusetts, Worcester, MA 01605, USA.ORCID 0000-0002-2219-5668
Gregory A NewbyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0001-7869-2615
Anton J BlatnikDepartment of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.ORCID 0000-0002-3557-9011
Aditya RaguramMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0002-5749-7308
Michelle F RichterMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0002-6113-6820
Kevin T ZhaoMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0003-3440-2094
Jonathan M LevyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0002-8061-7953
Max W ShenMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
W David ArnoldDepartment of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.ORCID 0000-0001-9889-7036
Dan WangHorae Gene Therapy Center, UMass Chan Medical School, University of Massachusetts, Worcester, MA 01605, USA.ORCID 0000-0001-9079-2360
Jun XieHorae Gene Therapy Center, UMass Chan Medical School, University of Massachusetts, Worcester, MA 01605, USA.ORCID 0000-0001-9565-1567
Guangping GaoHorae Gene Therapy Center, UMass Chan Medical School, University of Massachusetts, Worcester, MA 01605, USA.ORCID 0000-0003-0097-9012
Arthur H M BurghesDepartment of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.ORCID 0000-0001-9031-1555
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0002-9943-7557
Broad Institute · USUniversity of Massachusetts Chan Medical School · USThe Ohio State University Wexner Medical Center · USUniversity of Missouri Health System · US
Funding
VIROSOME/PROTEOLIPOSOME MEDIATED GENE TRANSFERP01HL053749 · NHLBI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI WEISS, MITCHELL J · 1994 to 2022
$43.6MCenter for Genomic Editing and Recording: Development and Application of Next-Generation Genome and Epigenome Editing Methods to Advance the Study and Treatment of Human DiseaseRM1HG009490 · NHGRI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI Brittany S. Adamson, Martin Joseph Ankrah Aryee · 2017 to 2026
$22.7MIntegrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4MGraduate Training in Computational and Systems BiologyT32GM087237 · NIGMS · MASSACHUSETTS INSTITUTE OF TECHNOLOGY · PI BURGE, CHRISTOPHER B · 2009 to 2023
$4.6MTherapy for Spinal Muscular AtrophyR01HD060586 · NICHD · OHIO STATE UNIVERSITY · PI BURGHES, ARTHUR H. M. · 2010 to 2021
$3.7MPrinciples of Scientific Citizenship: Ethics, Communication, and Leadership for ScientistsT32GM095450 · NIGMS · HARVARD UNIVERSITY · PI WALKER, SUZANNE · 2011 to 2020
$3.5MExpanding the Scope of Base EditingU01AI142756 · NIAID · BROAD INSTITUTE, INC. · PI LIU, DAVID R · 2018 to 2022
$2.1MContinuous Evolution of Proteins with Novel Therapeutic PotentialR01EB022376 · NIBIB · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2019
$1.8MIn vivo precision genome editing to correct genetic diseaseR00HL163805 · NHLBI · JOHNS HOPKINS UNIVERSITY · PI NEWBY, GREGORY A. · 2023 to 2025
$747kPrecision Base Editing for the Treatment of Motor Neuron DiseasesR00NS119743 · NINDS · BOSTON CHILDREN'S HOSPITAL · PI Mandana Arbab · 2023 to 2026
$740kPrecision Base Editing for the Treatment of Motor Neuron DiseasesK99NS119743 · NINDS · BROAD INSTITUTE, INC. · PI ARBAB, MANDANA · 2021 to 2022
$253kIn vivo precision genome editing to correct genetic diseaseK99HL163805 · NHLBI · BROAD INSTITUTE, INC. · PI NEWBY, GREGORY A. · 2022 to 2022
$167kHoward Hughes Medical InstituteNHGRI NIH HHS RM1 HG009490NHLBI NIH HHS K99 HL163805NHLBI NIH HHS P01 HL053749NHLBI NIH HHS R00 HL163805NIAID NIH HHS U01 AI142756NIBIB NIH HHS R01 EB022376NICHD NIH HHS R01 HD060586NIGMS NIH HHS R35 GM118062NIGMS NIH HHS T32 GM087237NIGMS NIH HHS T32 GM095450NINDS NIH HHS K99 NS119743NINDS NIH HHS R00 NS119743
Abstract
Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from survival motor neuron (SMN) protein insufficiency resulting from
Indexed as
Gene EditingMuscular Atrophy, SpinalSurvival of Motor Neuron 1 ProteinSurvival of Motor Neuron 2 ProteinAnimalsFibroblastsMiceMotor NeuronsSmn1 protein, mouseSMN2 protein, mouseSurvival of Motor Neuron 1 ProteinSurvival of Motor Neuron 2 Protein
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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.