公开(公告)号：US20210309999A1

公开(公告)日：2021-10-07

申请号：US17265489

申请日：2019-08-02

Applicant: Genzyme Corporation

Inventor： Bradford ELMER ,  Brenda RICHARDS ,  Martine LATTA-MAHIEU ,  Maria Carmen OBINU ,  Véronique TAUPIN ,  Véronique BLANCHARD

IPC: C12N15/113 ,  C12N15/86

Abstract: Provided herein are RNAi molecules for treating neurodegenerative synucleinopathies. In some embodiments, the RNAi molecules target expression of alpha-synuclein (SNCA). Further provided herein are expression constructs, vectors (e.g rAAV), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat neurodegenerative synucleinopathies including Parkinson's disease, multiple system atrophy, and dementia with Lewy bodies.