ArticleNature2018
Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents.
Registry-linked trialOpen access · greenAbstract read
In one paragraph
Article in Nature, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06729632 (Non-Invasive System to Deliver Therapeutic Hypothermia for Protection Against Noise-Induced Hearing Loss), which is not on this map. Cited by 318 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
318citing papers in PubMed, 1 pooled it
24.7field-weighted citation impact, top 1% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Non-Invasive System to Deliver Therapeutic Hypothermia for Protection Against Noise-Induced Hearing Loss
Who cites it
318 citing papers in PubMed, 1 synthesis or guideline pooled it, 534 citations in OpenAlex.
- Transduction Efficiency and Immunogenicity of Viral Vectors for Cochlear Gene Therapy: A Systematic Review of Preclinical Animal Studies.Frontiers in cellular neuroscience · 2021Pooled it
- The impact of technology on promoting physical activities and mental health: a gender-based study.BMC psychology · 2023Trial
- CRISPRing through time: How cutting-edge technology is revolutionizing life sciences and medicine.Molecular therapy. Nucleic acids · 2026Review
- Mapping the global trends and hotspots of research on gene therapy and hair cell regeneration for hearing loss: a comprehensive data-mining-based study.Gene therapy · 2026Article
- Single-cell and in vivo profiling reveal heterogeneous and organ-specific CRISPR-Cas9 off-target and translocation outcomes.Nature communications · 2026Article
- Mechanistic machine learning for prediction of prime editing outcomes.Nature biotechnology · 2026Article
- Gene Editing Technologies for Hereditary Hearing Loss: Prospects and Challenges.Neuroscience bulletin · 2026Review
- Long-term restoration of auditory function in a DFNA2 mouse model by adenine base editing.EMBO molecular medicine · 2026Article
- Physics-Informed Optimization for the Sub-Feature-Scale Fabrication of Hollow Microneedles via Digital Light Processing.Micromachines · 2026Article
- Stem cell-based and gene editing strategies in the treatment of congenital and acquired sensorineural hearing loss: a narrative review.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2026Review
- Non-viral delivery of genome-editing tools for treatment of genetic disorders.Acta pharmaceutica Sinica. B · 2026Review
- BEstimate: a computational tool for the design and interpretation of CRISPR base editing experiments.Genome biology · 2026Article
- Interaction-aware dexterous robot for minimally invasive transcanal inner ear interventions.Nature communications · 2026Article
- Allele-specific suppression of pathogenic bestrophin-1 transcripts by CRISPR/Cas9-mediated genome editing.Genome medicine · 2026Article
- DNA Repair Enzyme Regulation Strategy for Enhanced Pancreatic Neuroendocrine Tumor Therapy via Targeting siRNA-Lipid Nanoparticles.ACS nano · 2026Article
- Brain tumor detection based on transcription regulation features identified from public cerebrospinal fluid cell-free DNA sequencing data.Translational cancer research · 2026Article
- Mechanistic machine learning enables interpretable and generalizable prediction of prime editing outcomes.bioRxiv : the preprint server for biology · 2026Article
- CRISPR Base Editing Correction of TGFBI Mutations in Autosomal Dominant Corneal Dystrophies.Investigative ophthalmology & visual science · 2026Article
- Gene modification: Exploring the potential in treating kidney diseases.Pharmacological research · 2026Review
- Intermittent hypobaric pressure induces selective senescent cell death and alleviates age-related osteoporosis.Nature biomedical engineering · 2026Article
258 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
20 authors at 7 institutions in 2 countries.
Xue GaoDepartment of Chemistry and Chemical Biology, Harvard University, Cambridge, Massachusetts 02138, USA.
Yong TaoDepartment of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts 02114, USA.
Veronica LamasDepartment of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts 02114, USA.
Mingqian HuangDepartment of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts 02114, USA.
Wei-Hsi YehDepartment of Chemistry and Chemical Biology, Harvard University, Cambridge, Massachusetts 02138, USA.
Bifeng PanDepartments of Otolaryngology and Neurology, F.M. Kirby Neurobiology Center Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts 02115, USA.
Yu-Juan HuDepartment of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts 02114, USA.
Johnny H HuDepartment of Chemistry and Chemical Biology, Harvard University, Cambridge, Massachusetts 02138, USA.
David B ThompsonDepartment of Chemistry and Chemical Biology, Harvard University, Cambridge, Massachusetts 02138, USA.
Yilai ShuDepartment of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts 02114, USA.
Yamin LiDepartment of Biomedical Engineering, Tufts University, Medford, Massachusetts 02155, USA.
Hongyang WangDepartment of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts 02114, USA.
Shiming YangDepartment of Otolaryngology & Head Neck Surgery, Key Lab of Hearing Impairment Science of Ministry of Education, Key Lab of Hearing Impairment Prevention and Treatment of Beijing City, Chinese PLA Medical School, Beijing, China.
Qiaobing XuDepartment of Biomedical Engineering, Tufts University, Medford, Massachusetts 02155, USA.
Daniel B PolleyDepartment of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts 02114, USA.
M Charles LibermanDepartment of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts 02114, USA.
Wei-Jia KongDepartment of Otorhinolaryngology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei 430022, China.
Jeffrey R HoltDepartments of Otolaryngology and Neurology, F.M. Kirby Neurobiology Center Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts 02115, USA.
Zheng-Yi ChenDepartment of Otolaryngology and Program in Neuroscience, Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts 02114, USA.
David R LiuDepartment of Chemistry and Chemical Biology, Harvard University, Cambridge, Massachusetts 02138, USA.
Massachusetts Eye and Ear Infirmary · USHoward Hughes Medical Institute · USBoston Children's Hospital · USHarvard University · USHuazhong University of Science and Technology · CNTufts University · USBroad Institute · US
Funding
SINGLE-NEURON MARKING IN THE STUDY OF ABNORMAL COCHLEASR01DC000188 · NIDCD · MASSACHUSETTS EYE AND EAR INFIRMARY · PI Gabriel Corfas, M. Charles Liberman · 1989 to 2026
$15.1MImaging Core PlanP30DC005209 · NIDCD · MASSACHUSETTS EYE AND EAR INFIRMARY · PI ADAMS, JOE C · 2002 to 2016
$12.0MActivity-Dependent Influences on Auditory CircuitsR01DC009836 · NIDCD · VANDERBILT UNIVERSITY · PI POLLEY, DANIEL B. · 2009 to 2024
$6.7MIntegrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4MTMC gene function in sensory hair cellsR01DC013521 · NIDCD · BOSTON CHILDREN'S HOSPITAL · PI JEFFREY R HOLT · 2013 to 2026
$6.0MMouse Neurodevelopmental Behavior CoreU54HD090255 · NICHD · BOSTON CHILDREN'S HOSPITAL · PI POMEROY, SCOTT LOREN · 2016 to 2020
$5.2MHair Cell DevelopmentR01DC006908 · NIDCD · MASSACHUSETTS GENERAL HOSPITAL · PI CHEN, ZHENG-YI · 2005 to 2015
$2.7MContinuous Evolution of Proteins with Novel Therapeutic PotentialR01EB022376 · NIBIB · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2019
$1.8MPHYSICAL MAPPING OF ACADIAN USHER SYNDROMEF32DC000138 · NIDCD · OCHSNER CLINIC FOUNDATION · PI DOUCET, JOHN P · 1994 to 1995
–Howard Hughes Medical InstituteNIBIB NIH HHS R01 EB022376NICHD NIH HHS U54 HD090255NIDCD NIH HHS F32 DC000138NIDCD NIH HHS P30 DC005209NIDCD NIH HHS R01 DC000188NIDCD NIH HHS R01 DC006908NIDCD NIH HHS R01 DC009836NIDCD NIH HHS R01 DC013521NIGMS NIH HHS R35 GM118062
Abstract
Although genetic factors contribute to almost half of all cases of deafness, treatment options for genetic deafness are limited. We developed a genome-editing approach to target a dominantly inherited form of genetic deafness. Here we show that cationic lipid-mediated in vivo delivery of Cas9-guide RNA complexes can ameliorate hearing loss in a mouse model of human genetic deafness. We designed and validated, both in vitro and in primary fibroblasts, genome editing agents that preferentially disrupt the dominant deafness-associated allele in the Tmc1 (transmembrane channel-like gene family 1) Beethoven (Bth) mouse model, even though the mutant Tmc1
Indexed as
Acoustic StimulationAllelesAnimalsAnimals, NewbornAuditory ThresholdBase SequenceCell SurvivalCochleaCRISPR-Associated ProteinsCRISPR-Cas SystemsDisease Models, AnimalEvoked Potentials, Auditory, Brain StemFemaleFibroblastsGene EditingGenes, DominantCRISPR-Associated ProteinsLiposomesMembrane ProteinsTMC1 protein, mouse
Identifiers
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Read underepoch 390
Registered trials
NCT06729632ClinicalTrials.gov
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.