公开(公告)号：US20220033824A1

公开(公告)日：2022-02-03

申请号：US17297521

申请日：2019-11-27

申请人： University of Massachusetts

发明人： Christian Mueller ,  Gabriela Toro Cabrera ,  Robert H. Brown, JR.

IPC分类号： C12N15/113 ,  C12N15/86 ,  C12N15/10 ,  A61K35/761

摘要： The disclosure provides, in some aspects, compositions (e.g., isolated nucleic acids and rAAVs) comprising a transgene which encodes at least one inhibitory nucleic acid which decreases expression of a gene encoding a serine palmitoyltransferase protein (e.g., SPTLC1). In some aspects, the disclosure relates to methods of treating hereditary sensory and autonomic neuropathy 1 (HSAN1) by administering the compositions described by the disclosure to a cell or subject.