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US 2026/0191908 A1Published application
Compositions and methods for treating genetic disorders
OwnerJuan Antonio Bueren RONCERO, Paula Rio GALDO +6inventors
InventorsJuan Antonio Bueren RONCERO, Paula Rio GALDO, Susana Navarro Ordóñez, Julian Sevilla NAVARRO, Jose Carlos Segovia SANZ, Africa Gonzalez MURILLO, Jose Antonio Casado OLEA, Guillermo Güenechea AMURRIO
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Abstract
The present invention provides compositions and methods for rescuing FANCA expression in cells with diminished or no FANCA gene product. In particular, methods and compositions for treating genetic disorders are disclosed.
Claims28
(canceled)
An expression cassette comprising a polynucleotide sequence comprising in the following 5′ to 3′ order:
- (a) a human phosphoglycerate kinase (PGK) promoter sequence or a functional homolog or variant thereof;
- (b) a sequence encoding a human FANC polypeptide or a functional fragment or variant thereof;
- (c) a woodchuck hepatitis virus regulatory element (WPRE) sequence or a functional variant or fragment thereof,
- wherein the sequence encoding the human FANC polypeptide or functional fragment or variant thereof is operably linked to the PGK promoter sequence;
- and
- wherein the WPRE element comprises a sequence having at least 90% identity to SEQ ID NO:
- 23.
4 more elements
Dependencies are inferred from the claim text.