Title:
MODULATION OF SPTLC1 VIA RECOMBINANT ADENO-ASSOCIATED VECTORS
Document Type and Number:
WIPO Patent Application WO/2020/112967
Kind Code:
A8
Abstract:
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.
Inventors:
MUELLER CHRISTIAN (US)
CABRERA GABRIELA (US)
BROWN ROBERT (US)
CABRERA GABRIELA (US)
BROWN ROBERT (US)
Application Number:
PCT/US2019/063558
Publication Date:
July 30, 2020
Filing Date:
November 27, 2019
Export Citation:
Assignee:
UNIV MASSACHUSETTS (US)
International Classes:
C12N15/113; A61K35/76; A61P25/00; C12N15/86
Attorney, Agent or Firm:
BEYERSDORF, Matthew, S. et al. (US)
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