Gm3 synthase vectors and uses thereof
AssigneesUniversity of Massachusetts, The Clinic for Special Children, Inc.
InventorsHuiya Yang, Guangping Gao, Kevin A. Strauss, Dan Wang
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Abstract
Aspects of the disclosure relate to 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 by administering an rAAV expressing one or more Ganglioside GM3 synthase (GM3S) isoforms via intracerebroventricular (ICV) administration.
Claims32
A method for treating a GM3 synthase (GM3S) deficiency in a subject in need thereof, the method comprising administering to a subject having a GM3 synthase (GM3S) deficiency a recombinant adeno-associated virus (rAAV) via intracerebroventricular (ICV) administration, wherein the rAAV comprises:
- (i) a capsid protein;
- and
- (ii) an isolated nucleic acid comprising a transgene having a nucleic acid sequence encoding one or more monosialodihexosylganglioside synthase (GM3S) protein isoforms, wherein the transgene is flanked by adeno-associated virus (AAV) inverted terminal repeats (ITRs).
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A recombinant adeno associated virus (rAAV) comprising
- (i) an AAV Cap-Mac capsid protein;
- and
- (ii) an isolated nucleic acid comprising a transgene having a nucleic acid sequence encoding one or more monosialodihexosylganglioside synthase (GM3S) Ia Type 2 protein isoforms, and wherein the transgene is flanked by adeno-associated virus (AAV) inverted terminal repeats (ITRs).
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Dependencies are inferred from the claim text.