University of Florida Research Foundation; Incorporated
发明人:
Arun Srivastava,Chen Ling,Mavis Agbandje-McKenna
申请号：
US15552127
公开号：
US20180135074A1
申请日:
2016.02.19
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Provided herein are recombinant AAV (rAAV) particles comprising a nucleic acid vector comprising a parvovirus B 19p6 promoter operatively linked to a heterologous gene, such as a human globin gene, and rAAV capsid proteins comprising one or more amino acid substitutions in a surface exposed loop of the capsid protein that result, e.g., in increased P antigen binding compared to a corresponding un-mutated AAV capsid protein. Also provided are methods and compositions related to such capsid proteins, methods of targeting gene expression to a cell of erythroid lineage, methods of treating a hemoglobinopathy using such rAAV particles, and methods for efficient transduction of a host cell suspension with a rAAV.
