Delivery of proteins using adeno-associated virus (AAV) vectors

摘要

Disclosed herein are compositions, systems and methods for delivery of proteins of interest using adeno-associated virus (AAV) vectors.

主权项

1. A viral vector, comprising:
a 5′ inverted terminal repeat (ITR) of adeno-associated virus (AAV) and a 3′ AAV ITR; a synthetic promoter, wherein the promoter comprises a nucleotide sequence having at least 95% sequence identity to the sequence of SEQ ID NO: 1; a restriction site downstream of the promoter to allow insertion of a polynucleotide encoding one or more proteins of interest, and a posttranscriptional regulatory element downstream of the restriction site, wherein the promoter, the restriction site and the posttranscriptional regulatory element are located downstream of the 5′ AAV ITR and upstream of the 3′ AAV ITR.