ArticleNature2021
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice.
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Article in Nature, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 292 papers.
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292citing papers in PubMed
31.4field-weighted citation impact, top 1% of its field
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Who cites it
292 citing papers in PubMed, 474 citations in OpenAlex.
- Human trilayer engineered blood vessel reveals influence of fibroblasts on disease progression in model of Hutchinson-Gilford progeria.Science advances · 2026Article
- Beyond Precision: A Multidimensional Framework for Selecting Genetic Medicine Platforms.Cells · 2026Review
- Base editing for precision therapeutics.Cell genomics · 2026Review
- In Vivo Base Editing Partially Rescues Bone Dysplasia in a Mouse Model of Hutchinson-Gilford Progeria Syndrome.Aging cell · 2026Article
- Mutation-agnostic base editing of the progerin farnesylation site rescues Hutchinson-Gilford progeria syndrome phenotypes in neuromuscular organoids.Nature communications · 2026Article
- Mechanistic machine learning for prediction of prime editing outcomes.Nature biotechnology · 2026Article
- Review
- The cardiac 4D nucleome: nuclear and chromatin dynamics across development, disease and ageing.Nature reviews. Cardiology · 2026Review
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
- Reaching for the cure as progeria research comes of age at the 12th international scientific workshop.Nature aging · 2026Article
- Review
- Ameliorating calcium homeostasis improves longevity and healthspan in progeroid and naturally aged mice.Nature communications · 2026Article
- First Generation Proteolysis Targeting Chimeras (PROTACs) for the Treatment of Progeria.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- The senescence-inhibitory p53 isoform Δ133p53α: enhancing cancer immunotherapy and exploring novel therapeutic approaches for senescence-associated diseases.GeroScience · 2026Review
- Optimized lentivirus-derived virus-like particles for efficient delivery of Cas9-based genome editors.Nucleic acids research · 2026Article
- Designing genome editing experiments with EditABLE.Genome biology · 2026Article
- Spatial profiling of gene editing by in situ sequencing in mice and macaques.Nature biomedical engineering · 2026Article
- Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice.Nature biomedical engineering · 2026Article
- Increased Telomere Mobility in Progeria is Restored by Isoprenylcysteine Carboxyl Methyltransferase Inhibition.bioRxiv : the preprint server for biology · 2026Article
- In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression.Genome medicine · 2026Article
232 more citing papers are in PubMed but not listed here.
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Authors and funding
21 authors at 6 institutions in 2 countries.
Luke W Koblan *Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.ORCID http://orcid.org/0000-0003-2814-4412
Michael R Erdos *National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.ORCID http://orcid.org/0000-0002-6603-1833
Christopher WilsonMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.ORCID http://orcid.org/0000-0002-8012-2440
Wayne A CabralNational Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.ORCID http://orcid.org/0000-0002-0563-5289
Jonathan M LevyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.ORCID http://orcid.org/0000-0002-8061-7953
Zheng-Mei XiongNational Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
Urraca L TavarezNational Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.ORCID http://orcid.org/0000-0002-2577-5377
Lindsay M DavisonDivision of Cardiovascular Medicine, Vanderbilt University Medical Center, Nashville, TN, USA.
Yantenew G GeteDepartment of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD, USA.
Xiaojing MaoDepartment of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD, USA.
Gregory A NewbyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
Sean P DohertyDivision of Cardiovascular Medicine, Vanderbilt University Medical Center, Nashville, TN, USA.
Narisu NarisuNational Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.ORCID http://orcid.org/0000-0002-8483-1156
Quanhu ShengDepartment of Biostatistics, Vanderbilt University Medical Center, Nashville, TN, USA.
Chad KrilowNational Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
Charles Y LinDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.ORCID http://orcid.org/0000-0002-9155-090X
Leslie B GordonHasbro Children's Hospital, Alpert Medical School of Brown University, Providence, RI, USA.
Kan CaoDepartment of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD, USA.ORCID http://orcid.org/0000-0002-5806-2939
Francis S CollinsNational Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA. francis.collins3@nih.gov.ORCID http://orcid.org/0000-0002-1023-7410
Jonathan D BrownDivision of Cardiovascular Medicine, Vanderbilt University Medical Center, Nashville, TN, USA. jonathan.d.brown@vumc.org.ORCID http://orcid.org/0000-0002-0359-6167
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA. drliu@fas.harvard.edu.ORCID http://orcid.org/0000-0002-9943-7557
National Institutes of Health · USBroad Institute · USVanderbilt University Medical Center · USUniversity of Maryland, College Park · USBaylor College of Medicine · USBoston Children's Hospital · US
Funding
Translational Analysis CoreP30DK058404 · NIDDK · VANDERBILT UNIVERSITY MEDICAL CENTER · PI RICHARD M. PEEK · 2002 to 2026
$29.9MCenter 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
$22.7MHutchinson-Gilford Progeria syndrome--a model for the genetics of aging.ZIAHG200305 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI ERDOS, MICHAEL · 2009 to 2025
$18.1MIntegrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4MHutchinson-Gilford Progeria Syndrom--Genetic Aging ModelZ01HG200305 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI COLLINS, FRANCIS S. · 2003 to 2008
$2.6MEfficient in Vivo RNP-based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid NanoparticlesUG3TR002636 · NCATS · MASSACHUSETTS EYE AND EAR INFIRMARY · PI CHEN, ZHENG-YI, LIU, DAVID R · 2018 to 2020
$2.4MElucidating the chromatin-dependent mechanisms governing chronic inflammatory activation of endothelial cells in atherosclerosis.R01HL146654 · NHLBI · VANDERBILT UNIVERSITY MEDICAL CENTER · PI BROWN, JONATHAN DAVID · 2020 to 2023
$2.4MDelivery Technologies for In Vivo Genome EditingUG3AI150551 · NIAID · BETH ISRAEL DEACONESS MEDICAL CENTER · PI CHAIKOF, ELLIOT · 2019 to 2021
$2.3MInvestigation of smooth muscle cell loss in progeriaR01HL126784 · NHLBI · UNIV OF MARYLAND, COLLEGE PARK · PI CAO, KAN · 2016 to 2020
$2.1MExpanding 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
$1.8MIntramural NIH HHS Z01 HG200305NCATS NIH HHS UG3 TR002636NHGRI NIH HHS RM1 HG009490NHLBI NIH HHS R01 HL126784NHLBI NIH HHS R01 HL146654NIAID NIH HHS U01 AI142756NIAID NIH HHS UG3 AI150551NIBIB NIH HHS R01 EB022376NIDDK NIH HHS P30 DK058404NIGMS NIH HHS R35 GM118062
Abstract
Hutchinson-Gilford progeria syndrome (HGPS or progeria) is typically caused by a dominant-negative C•G-to-T•A mutation (c.1824 C>T; p.G608G) in LMNA, the gene that encodes nuclear lamin A. This mutation causes RNA mis-splicing that produces progerin, a toxic protein that induces rapid ageing and shortens the lifespan of children with progeria to approximately 14 years
Indexed as
MutationAdenineAllelesAlternative SplicingAnimalsAortaBase PairingChildDisease Models, AnimalDNAFemaleFibroblastsGene EditingHumansLamin Type ALongevityAdenineDNALamin Type ALMNA protein, humanMutant Proteinsprelamin ARNA
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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.