Gene therapy in succinic semialdehyde dehydrogenase deficiency (ssadhd)
AssigneeChildren’s Medical Center Corporation
InventorsAlexander Rotenberg, Hing Cheong Lee, Phillip Lawrence Pearl
Life of this patent
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Abstract
Provided herein are methods and compositions for treating succinic semialdehyde dehydrogenase deficiency (SSADHD) using gene therapy to restore, at least partially, expression and/or activity of succinic semialdehyde dehydrogenase (SSADH).
Claims25
A method of treating succinic semialdehyde dehydrogenase deficiency (SSADHD) in a subject, the method comprising administering to the subject an effective amount of a polynucleotide comprising a promoter sequence and a sequence encoding succinic semialdehyde dehydrogenase (SSADH).
A polynucleotide comprising a nucleotide sequence having at least 90%, at least 95%, at least 98%, at least 99% or 100% identity to SEQ ID NO:3, wherein the nucleotide sequence encodes for succinic semialdehyde dehydrogenase (SSADH).
Dependencies are inferred from the claim text.