Inventors:
- South San Francisco CA, US
Stephanie TAGLIATELA - San Francisco CA, US
Anne TANENHAUS - Brisbane CA, US
Andrew YOUNG - Redwood City CA, US
Szu-Ying CHEN - San Francisco CA, US
Chi ZHANG - Daly City CA, US
Stephanie MARTIN - San Francisco CA, US
David OBERKOFLER - San Francisco CA, US
Victoria WONG - South San Francisco CA, US
Jianmin LI - South San Francisco CA, US
International Classification:
A61K 48/00
C12N 9/14
A61K 38/46
C12N 15/86
A61P 1/16
Abstract:
Provided herein are nucleic acid molecules and vectors comprising variant copper transporting ATPase 2 (ATP7B) nucleic acid sequences. Such sequences have been optimized for expression in mammalian cells, liver cells, and/or from an adeno associated viral vector (AAV), including truncated and/or codon optimized variants. Also provided are viral vectors comprising such ATP7B variant nucleic acid sequences, and methods of use thereof for treating disorders associated with an ATP7B deficiency, such as Wilsons disease.