DB-OTO Gene Therapy for Inherited Deafness
DB-OTO gene therapy improved hearing in patients with <i>OTOF</i>-related deafness, enabling natural acoustic hearing and normalizing hearing sensitivity in 3 of 12 treated patients. (Funded by Regeneron Pharmaceuticals; ClinicalTrials.gov number, NCT05788536.).
Authors
- Robert Nash (ORCID: https://orcid.org/0000-0002-7861-3734)
- G. S. Wilson (ORCID: https://orcid.org/0000-0003-0025-3641)
- Aiden Eliot Shearer (ORCID: https://orcid.org/0000-0002-5324-4805)
- Tera Quigley
- Jonathon P. Whitton
- William J. Riggs (ORCID: https://orcid.org/0000-0002-5572-5645)
- Jay T. Rubinstein
- Lawrence R. Lustig (ORCID: https://orcid.org/0000-0001-6718-9838)
- Aris Baras (ORCID: https://orcid.org/0000-0002-6830-3396)
- Manohar Bance (ORCID: https://orcid.org/0000-0001-8050-3617)
- Akira Ishiyama (ORCID: https://orcid.org/0000-0002-1913-0812)
- Meghan C. Drummond
- Vassili Valayannopoulos (ORCID: https://orcid.org/0000-0003-3942-8234)
- Hazem E. Hassan (ORCID: https://orcid.org/0000-0002-8633-3636)
- C. Eduardo Corrales (ORCID: https://orcid.org/0000-0002-5464-9257)
- Bret J. Musser (ORCID: https://orcid.org/0000-0003-2949-211X)
- Leah R. Sabin (ORCID: https://orcid.org/0000-0003-1757-7415)
- Christos A. Kyratsous (ORCID: https://orcid.org/0000-0002-2596-2906)
- Ignacio del Castillo (ORCID: https://orcid.org/0000-0003-1188-3665)
- Manuel Manrique (ORCID: https://orcid.org/0000-0003-2105-7009)
- Evie C. Landry (ORCID: https://orcid.org/0000-0002-3564-0484)
- Hubert Löwenheim (ORCID: https://orcid.org/0000-0001-8200-8804)
- Cedric V. Pritchett (ORCID: https://orcid.org/0000-0001-7355-8302)
- Rubén Polo (ORCID: https://orcid.org/0000-0003-3941-4641)
- Gary Herman (ORCID: https://orcid.org/0000-0003-4433-6529)
- George D. Yancopoulos
- John H. Greinwald
- Daniela Carvalho (ORCID: https://orcid.org/0000-0002-7062-8843)
- Rong Liu (ORCID: https://orcid.org/0000-0001-5170-6474)
- Yanping Chen (ORCID: https://orcid.org/0000-0002-5224-1100)
- Christian Weber (ORCID: https://orcid.org/0000-0003-4610-8714)
- Steven Harvey (ORCID: https://orcid.org/0000-0002-9253-0484)
- Jeffery J. Anderson
- Susan C. Irvin
Institutions
- Brigham and Women's Hospital (US)
- Cincinnati Children's Hospital Medical Center (US)
- Boston Children's Hospital (US)
- NewYork–Presbyterian Hospital (US)
- Harvard University (US)
- Regeneron (United States) (US)
- Medical College of Wisconsin (US)
- Instituto Cajal (ES)
- University of Cambridge (GB)
- Columbia University Irving Medical Center (US)
- University of California San Diego (US)
- NanoAffix Science (United States) (US)
- Nemours Children's Health System (US)
- Seattle Children's Hospital (US)
- Centro de Investigación Biomédica en Red (ES)
- Instituto Ramón y Cajal de Investigación Sanitaria (ES)
- Clinica Universidad de Navarra (ES)
- Institute of Child Health (IN)
- Nemours Children's Clinic (US)
- University College London (GB)
- University of Cincinnati (US)
- University of Tübingen (DE)
- Universidad de Navarra (ES)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2025-10-12
- DOI
- https://doi.org/10.1056/nejmoa2400521
- Citations
- 19
- Primary Topic
- Hearing, Cochlea, Tinnitus, Genetics
- Type
- article
- Field-Weighted Citation Impact
- 27.83