公开(公告)号：US20200164008A1

公开(公告)日：2020-05-28

申请号：US16631692

申请日：2018-07-17

Applicant: SPARK THERAPEUTICS, INC.

Inventor： John Fraser WRIGHT ,  Marcus Eugene CARR, JR.

IPC: A61K35/76 ,  C12N15/86 ,  C07K14/005 ,  C07K16/08 ,  A61K35/16 ,  G01N33/68 ,  C12N9/22 ,  C12N15/11 ,  A61M1/36 ,  A61M1/34 ,  A61M1/16

Abstract: Provided are methods of treating a subject in need of treatment for a disease caused by a loss of function or activity of a protein. Also provided are methods of treating a subject in need of treatment for a disease caused by a gain of function, activity or expression, of a protein.