Gm3 synthase vectors and uses thereof
AssigneesUniversity of Massachusetts, The Clinic for Special Children, Inc.
InventorsGuangping Gao, Huiya Yang, Kevin A. Strauss, Dan Wang
Life of this patent
Estimating the term…
Abstract
Aspects of the disclosure relate to compositions and methods for expressing one or more Ganglioside GM3 synthase (GM3S) isoforms in a cell or subject. In some aspects, the disclosure relates to methods for treating GM3 synthase deficiency in a subject in need thereof.
Claims56
An isolated nucleic acid comprising:
- (i) a first region comprising a first adeno-associated virus (AAV) inverted terminal repeat (ITR);
- and
- (ii) a second region comprising a transgene having a nucleic acid sequence encoding one or more monosialodihexosylganglioside synthase (GM3S) protein isoforms.
The isolated nucleic acid 1, wherein the transgene encodes a one or more GM3S Ia isoforms.
The isolated nucleic acid 1, wherein the transgene encodes a one or more GM3S Ib isoforms.
An isolated nucleic acid comprising:
- (i) a first region comprising a first adeno-associated virus (AAV) inverted terminal repeat (ITR);
- and
- (ii) a second region comprising a transgene having a nucleic acid sequence encoding one or more monosialodihexosylganglioside synthase (GM3S) Ia Type 2 protein isoforms.
An isolated nucleic acid comprising the sequence set forth in any one of SEQ ID NOs:
- 1-7, and 11-38.
Dependencies are inferred from the claim text.