The United States of America; as represented by the Secretary; Department of Health and Human Serv
发明人:
Zhijian Wu,Anand Swaroop,Suddhasil Mookherjee,Suja Hiriyanna,Tiansen Li
申请号:
US15556746
公开号:
US20180043035A1
申请日:
2016.03.11
申请国别(地区):
US
年份:
2018
代理人:
摘要:
Disclosed are adeno-associated virus (AAV) vectors comprising a nucleotide sequence encoding RP2 or RPGR-ORF15 and related pharmaceutical compositions. Also disclosed are methods of treating or preventing X-linked retinitis pigmentosa, increasing photoreceptor number in a retina of a mammal, and increasing visual acuity of a mammal using the vectors and pharmaceutical compositions.