您的位置: 首页 > 农业专利 > 详情页

Adeno-associated virus variant capsids and methods of use thereof
专利权人:
4D MOLECULAR THERAPEUTICS INC.
发明人:
Kirn, David,Kotterman, Melissa,Schaffer, David
申请号:
NZ74839517
公开号:
NZ748395A
申请日:
2017.05.12
申请国别(地区):
NZ
年份:
2019
代理人:
摘要:
Provided herein are variant adeno-assoeiated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AA V virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
相关专利

意 见 箱

匿名:登录

个人用户登录

找回密码

第三方账号登录

忘记密码

个人用户注册

必须为有效邮箱
6~16位数字与字母组合
6~16位数字与字母组合
请输入正确的手机号码

信息补充