ArticleScience translational medicine2025
In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene.
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Article in Science translational medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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9citing papers in PubMed
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Who cites it
9 citing papers in PubMed.
- Nanomedicine beyond carriers - devices, cells & living therapeutics.Biomedical microdevices · 2026Review
- Cytosine base editing of LPA in transgenic mice averts large deletions.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- A synergistic approach to curative liver therapy: multiplex CRISPR-Cas12a and repair drive for alpha-1 antitrypsin deficiency (AATD).Annals of medicine and surgery (2012) · 2025Article
- Article
- Repair Drive improves gene editing in the liver.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- AAV-mediated genome editing is influenced by the formation of R-loops.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- AAV-mediated genome editing is influenced by the formation of R-loops.bioRxiv : the preprint server for biology · 2024Article
- Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome.Molecular therapy. Methods & clinical development · 2024Article
- Recent advances in various adeno-associated viruses (AAVs) as gene therapy agents in hepatocellular carcinoma.Virology journal · 2024Review
Corrections and comments
- Update of
Authors and funding
29 authors.
Marco De GiorgiDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0002-9823-1397
So Hyun ParkDepartment of Bioengineering, Rice University, Houston, TX 77030, USA.ORCID 0000-0003-3599-1687
Adam CastorenoAlnylam Pharmaceuticals Inc., Cambridge, MA 02142, USA.
Mingming CaoDepartment of Bioengineering, Rice University, Houston, TX 77030, USA.ORCID 0000-0002-4234-1175
Ayrea HurleyDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0002-8697-8610
Lavanya SaxenaDepartment of Bioengineering, Rice University, Houston, TX 77030, USA.ORCID 0000-0001-6083-4013
Marcel A ChuecosDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0002-1840-0948
Christopher J WalkeyDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0002-1471-0447
Alexandria M DoerflerDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0003-2297-6103
Mia N FurgursonDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0009-0002-7363-0938
M Cecilia LjungbergDepartment of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0003-0544-878X
Kalyani R PatelDepartment of Pathology, Texas Children's Hospital, Houston, TX 77030, USA.ORCID 0000-0003-2654-5386
Sarah HydeAlnylam Pharmaceuticals Inc., Cambridge, MA 02142, USA.
Tyler ChickeringAlnylam Pharmaceuticals Inc., Cambridge, MA 02142, USA.ORCID 0009-0006-9529-7258
Stephanie LefebvreAlnylam Pharmaceuticals Inc., Cambridge, MA 02142, USA.
Kelly WassarmanAlnylam Pharmaceuticals Inc., Cambridge, MA 02142, USA.
Patrick MillerAlnylam Pharmaceuticals Inc., Cambridge, MA 02142, USA.
June QinAlnylam Pharmaceuticals Inc., Cambridge, MA 02142, USA.
Mark K SchlegelAlnylam Pharmaceuticals Inc., Cambridge, MA 02142, USA.ORCID 0000-0002-0127-608X
Ivan ZlatevAlnylam Pharmaceuticals Inc., Cambridge, MA 02142, USA.ORCID 0000-0003-2797-3032
Jun HanDivision of Medical Sciences, University of Victoria, Victoria, BC V8P 5C2, Canada.ORCID 0000-0002-9477-8782
Christine BeetonDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0002-2500-5874
Rich Gang LiDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0001-9661-8304
Jong KimDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
James F MartinDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0002-7842-9857
Karl-Dimiter BissigDepartment of Pediatrics, Alice and Y. T. Chen Center for Genetics and Genomics, Division of Medical Genetics, Duke University, Durham, NC 27710, USA.ORCID 0000-0002-9577-9250
Vasant JadhavAlnylam Pharmaceuticals Inc., Cambridge, MA 02142, USA.ORCID 0000-0002-6666-0250
Gang BaoDepartment of Bioengineering, Rice University, Houston, TX 77030, USA.ORCID 0000-0001-5501-554X
William R LagorDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0002-1703-5125
Funding
Tumor BiologyP30CA125123 · NCI · BAYLOR COLLEGE OF MEDICINE · PI DAVID R ROWLEY · 2007 to 2026
$73.9MTissue Analysis & Molecular Imaging CoreP30DK056338 · NIDDK · BAYLOR COLLEGE OF MEDICINE · PI Geoffrey A Preidis · 2001 to 2026
$28.3MPitx2 in atrial fibrillationR01HL118761 · NHLBI · BAYLOR COLLEGE OF MEDICINE · PI MARTIN, JAMES F · 2014 to 2025
$6.1MGenetic Repair of Familial HypercholesterolemiaR01HL132840 · NHLBI · BAYLOR COLLEGE OF MEDICINE · PI LAGOR, WILLIAM RAYMOND · 2017 to 2025
$5.4MPhysiological Dissection of the Mevalonate PathwayR01DK124477 · NIDDK · BAYLOR COLLEGE OF MEDICINE · PI William Raymond Lagor, NOAH Freeman SHROYER · 2020 to 2026
$3.5MBCM-Rice resource for the analysis of somatic gene editing in miceU42OD026645 · OD · BAYLOR COLLEGE OF MEDICINE · PI DICKINSON, MARY E, HEANEY, JASON D. · 2018 to 2022
$3.5MCytoskeletal Control of Yap in Heart RegenerationR01HL169511 · NHLBI · BAYLOR COLLEGE OF MEDICINE · PI James F Martin · 2023 to 2026
$2.7MHBB gene-editing for treating sickle cell diseaseR01HL152314 · NHLBI · RICE UNIVERSITY · PI BAO, GANG · 2020 to 2023
$2.5MVelcro AAV Vector for tissue-specific delivery of genome editing reagents with enhanced cargo capacityUG3HL151545 · NHLBI · RICE UNIVERSITY · PI BAO, GANG, LAGOR, WILLIAM RAYMOND · 2019 to 2021
$2.3MReawakening cardiomyocyte proliferation from structural mediated quiescenceR01HL171574 · NHLBI · BAYLOR COLLEGE OF MEDICINE · PI James F Martin, Sean M Wu · 2024 to 2026
$1.9MTherapeutic pathway reprogramming for metabolic liver diseaseR01DK115461 · NIDDK · DUKE UNIVERSITY · PI BISSIG, KARL-DIMITER · 2018 to 2020
$1.1MShared Sequel II Systems at BCM HGSCS10OD028587 · OD · BAYLOR COLLEGE OF MEDICINE · PI GIBBS, RICHARD A · 2020 to 2020
$508kNCI NIH HHS P30 CA125123NHLBI NIH HHS R01 HL118761NHLBI NIH HHS R01 HL132840NHLBI NIH HHS R01 HL152314NHLBI NIH HHS R01 HL169511NHLBI NIH HHS R01 HL171574NHLBI NIH HHS UG3 HL151545NIDDK NIH HHS P30 DK056338NIDDK NIH HHS R01 DK115461NIDDK NIH HHS R01 DK124477NIH HHS S10 OD028587NIH HHS U42 OD026645
Abstract
Homology-directed repair (HDR)-based genome editing is an approach that could permanently correct a broad range of genetic diseases. However, its utility is limited by inefficient and imprecise DNA repair mechanisms in terminally differentiated tissues. Here, we tested Repair Drive, a platform technology for selectively expanding HDR-corrected hepatocytes in adult mice in vivo. Repair Drive involves transient conditioning of the liver by knocking down an essential gene,
Indexed as
Genes, EssentialGene TargetingHepatocytesAnimalsDNA RepairGene EditingHumansHydrolasesLiverMiceMice, Inbred C57BLTransgenesfumarylacetoacetaseHydrolases
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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.