Evidence map›Paper›PMID 32493795›Full record

ArticleScience translational medicine2020

In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness.

Wei-Hsi Yeh, Olga Shubina-Oleinik, Jonathan M Levy, Bifeng Pan, Gregory A Newby, Michael Wornow, Rachel Burt, Jonathan C Chen, Jeffrey R Holt, David R Liu

Open access · greenAbstract read
In one paragraph

Article in Science translational medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 118 papers.

0numbers the graph read from it
0cells of the map it votes in
118citing papers in PubMed
9.5field-weighted citation impact, top 1% of its field
1 · What the graph read from it

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.

2 · The registry

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

118 citing papers in PubMed, 176 citations in OpenAlex.

  1. Review
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  10. Article
  11. Treating Hearing Loss: From Cochlear Implantation to Gene Therapy.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Review
  12. Article
  13. Article
  14. Molecular therapy. Nucleic acids · 2025
    Review
  15. Article
  16. Rational Design of Inner Ear Drug Delivery Systems.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Review
  17. Article
  18. CRISPR-based functional genomics tools in vertebrate models.Experimental & molecular medicine · 2025
    Review
  19. Molecular Determinants of TMC Protein Biogenesis and Trafficking.International journal of molecular sciences · 2025
    Article
  20. Advancing precision ear medicine: leveraging animal models for disease insights and therapeutic innovations.Mammalian genome : official journal of the International Mammalian Genome Society · 2025
    Review

58 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors at 3 institutions in 2 countries.

Wei-Hsi YehMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0001-7507-8177
Olga Shubina-OleinikDepartment of Otolaryngology, F.M. Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0003-2081-1700
Jonathan M LevyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0002-8061-7953
Bifeng PanDepartment of Otolaryngology, F.M. Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.
Gregory A NewbyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0001-7869-2615
Michael WornowMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0003-2215-6527
Rachel BurtMurdoch Children's Research Institute, The Royal Children's Hospital, Parkville, VIC 3052, Australia.
Jonathan C ChenMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.ORCID 0000-0003-4346-0501
Jeffrey R HoltDepartment of Otolaryngology, F.M. Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA. jeffrey.holt@childrens.harvard.edu drliu@fas.harvard.edu.ORCID 0000-0002-7182-8011
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA. jeffrey.holt@childrens.harvard.edu drliu@fas.harvard.edu.ORCID 0000-0002-9943-7557
Broad Institute · USBoston Children's Hospital · USRoyal Children's Hospital · AU

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
$22.7M
Integrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4M
TMC gene function in sensory hair cellsR01DC013521 · NIDCD · BOSTON CHILDREN'S HOSPITAL · PI JEFFREY R HOLT · 2013 to 2026
$6.0M
Mouse Neurodevelopmental Behavior CoreU54HD090255 · NICHD · BOSTON CHILDREN'S HOSPITAL · PI POMEROY, SCOTT LOREN · 2016 to 2020
$5.2M
Efficient 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
$2.4M
Expanding the Scope of Base EditingU01AI142756 · NIAID · BROAD INSTITUTE, INC. · PI LIU, DAVID R · 2018 to 2022
$2.1M
Howard Hughes Medical InstituteNCATS NIH HHS UG3 TR002636NHGRI NIH HHS RM1 HG009490NIAID NIH HHS U01 AI142756NICHD NIH HHS U54 HD090255NIDCD NIH HHS R01 DC013521NIGMS NIH HHS R35 GM118062
6 · The paper itself

Abstract

Most genetic diseases arise from recessive point mutations that require correction, rather than disruption, of the pathogenic allele to benefit patients. Base editing has the potential to directly repair point mutations and provide therapeutic restoration of gene function. Mutations of transmembrane channel-like 1 gene (

Indexed as

DeafnessMembrane ProteinsAnimalsFibroblastsHair Cells, AuditoryHearingHumansMiceMembrane ProteinsTMC1 protein, mouse

Identifiers

PMID32493795
PMCPMC8167884
OpenAlexW3032052376

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

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.