公开(公告)号：US20230287366A1

公开(公告)日：2023-09-14

申请号：US18119201

申请日：2023-03-08

Applicant: Genzyme Corporation

Inventor： Marco A. PASSINI ,  Robin J. ZIEGLER ,  James C. DODGE ,  Lamya SHIHABUDDIN ,  Seng H. CHENG

IPC: C12N9/16 ,  C12N15/85 ,  A61K48/00 ,  C12N15/86

CPC classification number: C12N9/16 ,  C12N15/8509 ,  A61K48/0083 ,  C12N15/86 ,  C12N2840/007 ,  C12N2750/14143 ,  A01K2207/00 ,  A01K2227/105 ,  G01N2500/00 ,  A01K2267/0306

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).

公开(公告)号：US20200080066A1

公开(公告)日：2020-03-12

申请号：US16544735

申请日：2019-08-19

Applicant: Genzyme Corporation

Inventor： Marco A. PASSINI ,  Robin J. ZIEGLER ,  James C. DODGE ,  Lamya SHIHABUDDIN ,  Seng H. CHENG

IPC: C12N9/16 ,  C12N15/86 ,  A61K48/00 ,  C12N15/85

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).