The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Aug. 19, 2025
Filed:
Nov. 27, 2019
Applicant:
University of Massachusetts, Boston, MA (US);
Inventors:
Christian Mueller, Worcester, MA (US);
Gabriela Toro Cabrera, Worcester, MA (US);
Robert H. Brown, Jr., Worcester, MA (US);
Assignee:
University of Massachusetts, Westborough, MA (US);
Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); A61K 35/761 (2015.01); C12N 15/10 (2006.01); C12N 15/86 (2006.01);
U.S. Cl.
CPC ...
C12N 15/1137 (2013.01); A61K 35/761 (2013.01); C12N 15/102 (2013.01); C12N 15/86 (2013.01); C12N 2310/141 (2013.01); C12N 2310/531 (2013.01); C12N 2750/14133 (2013.01); C12N 2750/14143 (2013.01); C12Y 203/0105 (2013.01);
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.