CODON-OPTIMIZED ABCB11 TRANSGENE FOR THE TREATMENT OF PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS TYPE 2 (PFIC2)

摘要

The present disclosure relates to gene therapy vector for use in the treatment of progressive familial intrahepatic cholestasis type 2. More specifically, the present invention relates to an adeno-associated virus vector comprising codon-optimized sequence encoding for the BSEP for the treatment of PFIC2.