ArticleNature communications2023
Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo.
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Article in Nature communications, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
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31citing papers in PubMed
6.9field-weighted citation impact, top 2% of its field
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Who cites it
31 citing papers in PubMed, 45 citations in OpenAlex.
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- A combinatorial synthetic strategy for developing genome-editing protein-delivery agents targeting mouse retina.Nature communications · 2026Article
- Antisense oligonucleotide therapy mitigates autosomal dominant progressive hearing loss in a murine model of human DFNA2.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Integrating CRISPR/Cas technology with clinical trials: Principles, progress and challenges.Asian journal of pharmaceutical sciences · 2025Review
- 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
- Study Models for Non-Syndromic Hearing Loss.Cells · 2025Review
- Single-dose genome editing therapy rescues auditory and vestibular functions in adult mice with DFNA41 deafness.The Journal of clinical investigation · 2025Article
- Gene therapy restores auditory function and rescues damaged inner hair cells in an aged Vglut3 knockout mouse model.Gene therapy · 2025Article
- Recapitulating dengue virus infection with human pluripotent stem cell-derived liver organoids for antiviral screening.Nature communications · 2025Article
- PAM-flexible adenine base editing rescues hearing loss in a humanized MPZL2 mouse model harboring an East Asian founder mutation.Nature communications · 2025Article
- Rational Design of Inner Ear Drug Delivery Systems.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Review
- The rise of cochlear gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Multicargo Porous Cochlear Electrode Coating for Antifibrosis After Cochlear Implantation.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 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
- Inner Ear Gene Therapy: An Overview from Bench to Bedside.Molecular diagnosis & therapy · 2025Review
- Innovative treatment of age-related hearing loss using MSCs and EVs with Apelin.Cell biology and toxicology · 2025Article
Corrections and comments
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Authors and funding
19 authors at 7 institutions in 2 countries.
Yong Tao *Department 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.
Veronica Lamas *Department 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.
Wan Du *Department 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.
Wenliang Zhu *Department 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.
Yiran LiDepartment 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.
Madelynn N WhittakerCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA, 02114, USA.ORCID http://orcid.org/0000-0002-3382-1134
John A ZurisMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
David B ThompsonMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
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 http://orcid.org/0000-0001-8599-569X
Yilai ShuDepartment 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 http://orcid.org/0000-0001-8893-434X
Xue GaoMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.ORCID http://orcid.org/0000-0003-3213-9704
Johnny H HuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Charles PeiEaton-Peabody Laboratory, Massachusetts Eye and Ear Infirmary, 243 Charles St., Boston, MA, 02114, USA.
Wei-Jia KongDepartment of Otorhinolaryngology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, China.ORCID http://orcid.org/0000-0001-5410-2297
Xuezhong LiuDepartment of Otolaryngology, University of Miami Miller School of Medicine, Miami, FL, USA.
Hao WuDepartment of Otolaryngology-Head and Neck Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200011, China.
Benjamin P KleinstiverCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA, 02114, USA.ORCID http://orcid.org/0000-0002-5469-0655
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA. dliu@broadinstitute.org.
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. Zheng-Yi_Chen@meei.harvard.edu.ORCID http://orcid.org/0000-0002-1452-4193
Massachusetts Eye and Ear Infirmary · USBroad Institute · USHarvard University · USMassachusetts General Hospital · USShanghai Jiao Tong University · CNUnion Hospital · CNUniversity of Miami · US
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
$7.2MIntegrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4MSCGE 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
$2.4MDelivery Technologies for In Vivo Genome EditingUG3AI150551 · NIAID · BETH ISRAEL DEACONESS MEDICAL CENTER · PI CHAIKOF, ELLIOT · 2019 to 2021
$2.3MExpanding the Scope of Base EditingU01AI142756 · NIAID · BROAD INSTITUTE, INC. · PI LIU, DAVID R · 2018 to 2022
$2.1MNCATS NIH HHS UG3 TR002636NCATS NIH HHS UH3 TR002636NHGRI NIH HHS RM1 HG009490NHGRI NIH HHS U24 HG010423NIAID NIH HHS U01 AI142756NIAID NIH HHS UG3 AI150551NIDCD NIH HHS R01 DC005575NIDCD NIH HHS R01 DC012115NIDCD NIH HHS R01 DC016875NIDCD NIH HHS R01 DC019404NIGMS NIH HHS R35 GM118062
Abstract
Mutations in Atp2b2, an outer hair cell gene, cause dominant hearing loss in humans. Using a mouse model Atp2b2
Indexed as
DeafnessHearing LossCRISPR-Cas SystemsHumansLiposomesRibonucleoproteinsRNA, Guide, CRISPR-Cas SystemsLiposomesRibonucleoproteinsRNA, Guide, CRISPR-Cas Systems
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