Novel <i>Grm6</i> Variant in a <i>no b-wave (nob)</i> Mouse Model: Phenotype Characterization and Gene Therapy
We identified a new nob mouse model that mimics the CSNB phenotype in human patients. Whereas gene therapy successfully restored mGluR6 expression, functional improvements were limited. Early treatment using a specific promoter is critical, and increasing transduction efficiency may improve gene therapy strategies.
Authors
- Nan‐Kai Wang (ORCID: https://orcid.org/0000-0002-6277-9879)
- Stephen H. Tsang (ORCID: https://orcid.org/0000-0001-9082-2427)
- Neoklis Makrides
- Melina A. Agosto (ORCID: https://orcid.org/0000-0002-3647-1926)
- Eugene Yu‐Chuan Kang (ORCID: https://orcid.org/0000-0001-6814-6530)
- Pei‐Hsuan Lin (ORCID: https://orcid.org/0000-0002-0855-5559)
- Jiaxu Wang (ORCID: https://orcid.org/0000-0001-7101-8230)
- Janet R. Sparrow (ORCID: https://orcid.org/0000-0001-7448-5950)
- John Peregrin
- Tai‐De Li (ORCID: https://orcid.org/0000-0003-0515-5135)
- Yun-Ju Tseng
- Winston Lee (ORCID: https://orcid.org/0000-0002-1777-8519)
- Theresa C. Swayne (ORCID: https://orcid.org/0000-0002-1745-9753)
- Emmet A. Sherman (ORCID: https://orcid.org/0000-0002-4891-9740)
- Xin Zhang (ORCID: https://orcid.org/0000-0001-5555-0825)
- Peiliang Wu (ORCID: https://orcid.org/0000-0002-6113-0779)
Institutions
- The Graduate Center, CUNY (US)
- City College of New York (US)
- Dalhousie University (CA)
- National Taiwan University (TW)
- Chang Gung University (TW)
- George Washington University (US)
- Columbia University Irving Medical Center (US)
- Chang Gung Memorial Hospital (TW)
- CUNY Advanced Science Research Center (US)
- National Yunlin University of Science and Technology (TW)
- Columbia University (US)
- National Yang Ming Chiao Tung University (TW)
Publication Details
- Journal
- Investigative Ophthalmology & Visual Science
- Published
- 2025-09-09
- DOI
- https://doi.org/10.1167/iovs.66.12.20
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00