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US 12,589,167 B2Granted
AAV gene therapy vector with podocyte-specific promoter
AssigneesThe University of Bristol, Syncona IP Holdco (3) Limited
InventorsDominic Schmidt, Valeryia Kuzmuk, Moin Saleem, Gavin Welsh
Life of this patent
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Abstract
A viral vector, wherein the viral vector comprises a COL4A3, COL4A4 or COL4A5 transgene.
Claims28
A viral vector, wherein the viral vector comprises a COL4A3, COL4A4 or COL4A5 transgene and a podocyte-specific promoter, wherein the podocyte-specific promoter is a minimal nephrin promoter NPHS1,
- wherein the minimal nephrin promoter NPHS1 comprises the nucleotide sequence shown as SEQ ID NO:
- 10, or a variant that is at least 70% identical to SEQ ID NO:
- 10, and
- wherein the minimal nephrin promoter has a length of about 0.6 kb or less.
1 more element
Dependencies are inferred from the claim text.