ArticleScience translational medicine2024
Targeted genome editing restores auditory function in adult mice with progressive hearing loss caused by a human microRNA mutation.
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Article in Science translational medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 1 of them a synthesis that pooled it.
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17citing papers in PubMed, 1 pooled it
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Who cites it
17 citing papers in PubMed, 1 synthesis or guideline pooled it.
- From cellular regeneration toward functional integration: a 15-year bibliometric analysis of inner ear hair cell regeneration (2011-2025).Frontiers in cellular neuroscience · 2026Pooled it
- Gene supplementation with precise transgene expression rescues hearing loss in a mouse model with an Mpzl2 East Asian founder variant.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Engineered Inner Ear Drug Delivery Systems for Hearing Loss Treatment.Research (Washington, D.C.) · 2026Review
- Evolutionary constraints impose multiple checkpoints that block mammalian sensory hair cell regeneration.Frontiers in cell and developmental biology · 2026Review
- Mechanisms of and mitigating strategies for cellular immune responses to CRISPR-associated nucleases in genome editing therapy.Frontiers in medicine · 2026Review
- Recent preclinical and clinical advances in gene therapy for hereditary hearing loss.Molecules and cells · 2025Review
- Extracellular vesicle-mediated gene editing for the treatment of nonsyndromic progressive hearing loss in adult mice.Science translational medicine · 2025Article
- Single-dose genome editing therapy rescues auditory and vestibular functions in adult mice with DFNA41 deafness.The Journal of clinical investigation · 2025Article
- Engineering adeno-associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing.Biomaterials · 2025Review
- Single-cell profiling identifies hair cell SLC35F1 deficiency as a signature of primate cochlear aging.Nature aging · 2025Article
- Recapitulating dengue virus infection with human pluripotent stem cell-derived liver organoids for antiviral screening.Nature communications · 2025Article
- Advancing precision ear medicine: leveraging animal models for disease insights and therapeutic innovations.Mammalian genome : official journal of the International Mammalian Genome Society · 2025Review
- Therapeutic restoration of miR-96 prevents hearing loss in mice through modulation of noise-induced and genetic pathways.iScience · 2025Article
- High-Throughput Screening to Identify Novel Compounds Affecting the Genome Editing Efficiency of CRISPR System.Molecules (Basel, Switzerland) · 2025Article
- Inner Ear Gene Therapy: An Overview from Bench to Bedside.Molecular diagnosis & therapy · 2025Review
- Human recombinant tyrosinase destabilization caused by the double mutation R217Q/R402Q.Protein science : a publication of the Protein Society · 2025Article
- Pathological mechanisms and candidate therapeutic approaches in the hearing loss of mice carrying human MIR96 mutations.Genome medicine · 2024Article
Corrections and comments
- Update of
Authors and funding
12 authors.
Wenliang ZhuDepartment of Otolaryngology-Head and Neck Surgery, Graduate Program in Speech and Hearing Bioscience and Technology and Program in Neuroscience, Harvard Medical School, Boston, MA 02115, USA.ORCID 0009-0006-8746-5346
Wan DuDepartment of Otolaryngology-Head and Neck Surgery, Graduate Program in Speech and Hearing Bioscience and Technology and Program in Neuroscience, Harvard Medical School, Boston, MA 02115, USA.ORCID 0009-0004-2427-6172
Arun Prabhu RameshbabuDepartment of Otolaryngology-Head and Neck Surgery, Graduate Program in Speech and Hearing Bioscience and Technology and Program in Neuroscience, Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0001-8599-569X
Ariel Miura ArmstrongDepartment of Otolaryngology-Head and Neck Surgery, Graduate Program in Speech and Hearing Bioscience and Technology and Program in Neuroscience, Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0002-1421-7791
Stewart SilverDepartment of Otolaryngology-Head and Neck Surgery, Graduate Program in Speech and Hearing Bioscience and Technology and Program in Neuroscience, Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0001-7411-2214
Yehree KimDepartment of Otolaryngology-Head and Neck Surgery, Graduate Program in Speech and Hearing Bioscience and Technology and Program in Neuroscience, Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0002-3056-198X
Wei WeiDepartment of Otolaryngology-Head and Neck Surgery, Graduate Program in Speech and Hearing Bioscience and Technology and Program in Neuroscience, Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0003-2036-7312
Yilai ShuENT Institute and Otorhinolaryngology Department of Eye & ENT Hospital, State Key Laboratory of Medical Neurobiology and MOE Frontiers Center for Brain Science, Fudan University, Shanghai 200031, China.ORCID 0000-0001-8893-434X
Xuezhong LiuDepartment of Otolaryngology, University of Miami School of Medicine, Miami, FL 33136, USA.
Morag A LewisWolfson Sensory, Pain and Regeneration Centre, King's College London, London WC2R 2LS, UK.ORCID 0000-0002-1846-8600
Karen P SteelWolfson Sensory, Pain and Regeneration Centre, King's College London, London WC2R 2LS, UK.ORCID 0000-0002-8019-5359
Zheng-Yi ChenDepartment of Otolaryngology-Head and Neck Surgery, Graduate Program in Speech and Hearing Bioscience and Technology and Program in Neuroscience, Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0002-1452-4193
Funding
Center 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
$22.7MDissemination and Coordinating Center for the SCGE ConsortiumU24HG010423 · NHGRI · MEDICAL COLLEGE OF WISCONSIN · PI DWINELL, MELINDA R · 2019 to 2023
$15.0MMolecular Genetics on Non Syndromic Hearing Loss (NSHL)R01DC005575 · NIDCD · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI LIU, XUE Z · 2001 to 2024
$10.6MSensory disorders in COVID -19 as an early identifier and healthcare worker protectionR01DC012115 · NIDCD · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI LIU, XUE Z · 2013 to 2022
$7.2MSCGE Disease Models Studies Supplement: Development of LNP-mediated based editing to treat Leber Congenital Amaurosis (LCA) for vision restoration in mouse modelUH3TR002636 · NCATS · MASSACHUSETTS EYE AND EAR INFIRMARY · PI CHEN, ZHENG-YI, LIU, DAVID R · 2021 to 2022
$3.8MDevelopment of Genome Editing as Treatment for Genetic Hearing LossR01DC016875 · NIDCD · MASSACHUSETTS EYE AND EAR INFIRMARY · PI CHEN, ZHENG-YI · 2019 to 2023
$3.0MDevelopment of CRISPR/Cas9-based exon-skipping strategies for the treatment of USH-associated deafnessR01DC019404 · NIDCD · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI SUSAN HALLORAN BLANTON, Zheng-Yi Chen · 2022 to 2026
$2.8MEfficient 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
$2.4MNCATS NIH HHS UG3 TR002636NCATS NIH HHS UH3 TR002636NHGRI NIH HHS RM1 HG009490NHGRI NIH HHS U24 HG010423NIDCD NIH HHS R01 DC005575NIDCD NIH HHS R01 DC012115NIDCD NIH HHS R01 DC016875NIDCD NIH HHS R01 DC019404Wellcome Trust
Abstract
Mutations in
Indexed as
DependovirusGene EditingHearing LossMicroRNAsMutationAnimalsBase SequenceCochleaCRISPR-Cas SystemsGenetic TherapyHearingHumansMiceRNA, Guide, CRISPR-Cas SystemsMicroRNAsMirn96 microRNA, mouseRNA, Guide, CRISPR-Cas Systems
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LicenceCC BY
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.