US Patent:
20200080066, Mar 12, 2020
Inventors:
- Cambridge MA, US
Robin J. ZIEGLER - Bridgewater NJ, US
James C. DODGE - Bridgewater NJ, US
Lamya SHIHABUDDIN - Brighton MA, US
Seng H. CHENG - Natick MA, US
International Classification:
C12N 9/16
C12N 15/86
A61K 48/00
C12N 15/85
Abstract:
This disclosure pertains to methods and compositions for tolerizing a mammal's brain to exogenously administered acid sphingomyelinase polypeptide by first delivering an effective amount of a transgene encoding the polypeptide to the mammal's hepatic tissue and then administering an effective amount of the transgene to the mammal's central nervous system (CNS).