Modulation of sptlc1 via recombinant adeno-associated vectors
AssigneeUniversity of Massachusetts
InventorsChristian Mueller, Gabriela Toro Cabrera, Robert H. Brown, JR.
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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.
Claims54
An isolated nucleic acid comprising a transgene encoding at least one inhibitory nucleic acid, wherein the at least one inhibitory nucleic acid reduces expression of a gene encoding a serine palmitoyltransferase subunit, and wherein the transgene is flanked by inverted terminal repeats (ITRs) derived from adeno-associated virus (AAV).
A recombinant AAV (rAAV) comprising:
- (i) an isolated nucleic acid comprising a transgene encoding an inhibitory nucleic acid, wherein the inhibitory nucleic acid reduces expression of a gene encoding a serine palmitoyltransferase subunit and wherein the transgene is flanked by inverted terminal repeats (ITRs) derived from adeno-associated virus (AAV), and
- (ii) at least one capsid protein.
Dependencies are inferred from the claim text.