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OwnerFrank J.T. STAAL, Karin PIKE-OVERZET +3inventors
InventorsFrank J.T. STAAL, Karin PIKE-OVERZET, Laura GARCIA-PEREZ, Marja C.J.A. VAN EGGERMOND, Arjan C. LANKESTER
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The present invention provides novel expression cassettes, retroviral plasmids, vectors, virions, compositions and recombinant cells comprising a promoter operably linked to a codon optimised recombination activating (RAG1) transgene. These novel expression cassettes, retroviral plasmids, vectors, virions, compositions and recombinant cells are useful in the treatment of diseases caused by complete or partial loss-of-function of the protein encoded by the rag-1 gene, such as RAG-deficient severe combined immunodeficiency (RAG1-SCID), Omenn Syndrome (OS), atypical-SCID or combined immunodeficiency (CID). Corresponding methods of treatment are also provided.
An expression cassette comprising a promoter operably linked to a RAG1 transgene that comprises the nucleic acid sequence of SEQ ID NO:
The expression cassette of any preceding claim, wherein the promoter is MND.
The expression cassette of any preceding claim, wherein the expression cassette further comprises a nucleotide sequence encoding Woodchuck hepatitis virus (WHP) posttranscriptional regulatory element (WPRE).
A retroviral plasmid comprising an expression cassette as claimed in any preceding claim.
Dependencies are inferred from the claim text.