18835452. GJB2 REGULATORY ELEMENTS AND USES THEREOF (Regeneron Pharmaceuticals, Inc.)
GJB2 REGULATORY ELEMENTS AND USES THEREOF
Organization Name
Regeneron Pharmaceuticals, Inc.
Inventor(s)
Kathryn Ellis of Arlington MA US
Gabriela Pregernig of Cambridge MA US
Meghan Drummond Samuelson of Katonah NY US
Sarah Cancelarich of Bloomingdale NJ US
Leah Sabin of Goldens Bridge NY US
Daniela Di Battista Miani of Yorktown Heights NY US
GJB2 REGULATORY ELEMENTS AND USES THEREOF
This abstract first appeared for US patent application 18835452 titled 'GJB2 REGULATORY ELEMENTS AND USES THEREOF
Original Abstract Submitted
The disclosure provides gap junction protein beta 2 (GJB2) promoters and enhancers, as well as vectors containing the same, that can be used to express an expression product in GJB2-expressing cells, including cochlear supporting cells. The GJB2 promoters and enhancers described herein may be operably linked to a polynucleotide, such as a transgene encoding a wild-type form of Gjb2, for treatment of subjects having or at risk of developing hearing loss (e.g., GJB2-related hearing loss).
- Regeneron Pharmaceuticals, Inc.
- Joseph Burns of Newton MA US
- Kathryn Ellis of Arlington MA US
- Tyler Gibson of Boston MA US
- Kevin Lebo of Weymouth MA US
- Gabriela Pregernig of Cambridge MA US
- Meghan Drummond Samuelson of Katonah NY US
- Sarah Cancelarich of Bloomingdale NJ US
- Leah Sabin of Goldens Bridge NY US
- Daniela Di Battista Miani of Yorktown Heights NY US
- A61K48/00
- A61K9/00
- A61K38/17
- A61P27/16
- C12N15/86
- CPC A61K48/0058