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US 12,553,036 B2Granted
GM3 synthase vectors and uses thereof
AssigneesUniversity of Massachusetts, The Clinic for Special Children, Inc.
InventorsGuangping Gao, Huiya Yang, Kevin A. Strauss, Dan Wang
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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.
Claims9
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 comprising a nucleic acid sequence that consists of the nucleotide sequence set forth in SEQ ID NO:
- 43 and encodes a monosialodihexosylganglioside synthase (GM3S) Ia Type 2 protein isoform.
1 more element
Dependencies are inferred from the claim text.