Back to results
US 12,630,839 B2Granted
Compositions for the treatment of Fabry disease
AssigneeSangamo Therapeutics, Inc.
InventorMarshall W. Huston
Life of this patent
Estimating the term…
Abstract
The present disclosure provides expression constructs comprising a GLA transgene encoding the at least one α-Gal A protein for use in expressing α-Gal A proteins and preventing, inhibiting or treating Fabry disease or one or more symptoms associated with Fabry disease.
Claims20
An adeno-associated virus (AAV) expression construct comprising an apolipoprotein E (APOE) enhancer comprising the nucleotide sequence as set forth in SEQ ID NO:
- 2 operably linked to an alpha 1-antitrypsin (hAAT) promoter comprising the nucleotide sequence as set forth in SEQ ID NO:
- 3, a human hemoglobin beta (HBB)-IGG intron comprising the nucleotide sequence as set forth in SEQ ID NO:
- 4, an α-galactosidase A (α-Gal A)transgene comprising the nucleotide sequence as set forth in SEQ ID NO:
- 5, a mutated Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element sequence comprising the nucleotide sequence as set forth in SEQ ID NO:
- 6, and a bovine growth hormone poly A signal sequence comprising the nucleotide sequence as set forth in SEQ ID NO:
- 7.
3 more elements
An adeno-associated virus (AAV) expression construct comprising the nucleotide sequence as set forth in SEQ ID NO:
- 9.
Dependencies are inferred from the claim text.