GENE THERAPY FOR TREATING CITRULLENEMIA
Application
US20260078405A1
Kind: A1
Mar 19, 2026
Assignee
The Trustees of the University of Pennsylvania
Inventors
James M. Wilson, Jenny Agnes Sidrane, Lili Wang
Abstract
Compositions and regimens useful in treating type I citrullenemia are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human Argininosuccinate Synthase 1 (ASS1).
CPC Classifications
C12N 15/86
A61P 43/00
C12Y 603/04005
A61K 48/00
C12N 2750/14143
C12N 2830/008
C12N 2830/42
Filing Date
2025-08-11
Application No.
19296641