OPTIMISED RAG1 DEFICIENT GENE THERAPY
Abstract
The present invention provides novel expression cassettes, retroviral plasmids, vectors, virions, compositionsand recombinant cellscomprising a promoter operably linked to a codon optimised recombination activating (RAG1)transgene. These novel expression cassettes, retroviral plasmids, vectors, virions, compositionsand recombinant cellsare useful in the treatment of diseases caused by complete or partial loss-of-function of the protein encoded by the rag-1gene, 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.
Inventors 15
- Staal, Dhr. Frank J.t.
- Pike-Overzet, Mw. Karin
- Garcia-Perez, Mw. Laura
- van Eggermond, Mw. Marja C.j.a.
- Lankester, Dhr. Arjan C.
- Frank J. T. Staal Aachen author as filed: Staal, Frank J.T.
- Karin Pike‐Overzet Aachen author as filed: Pike-Overzet, Karin
- Laura García-Pérez Aachen author as filed: Garcia-Perez, Laura
- Marja C.J.A. van Eggermond Aachen author as filed: van Eggermond, Marja C.J.A.
- Arjan C. Lankester Aachen author as filed: Lankester, Arjan C.
- Frank, J T Staal
- Karin, Pike-Overzet
- Laura, Garcia-Perez
- Marja, C J A van Eggermond
- Arjan, C Lankester
Inventors are linked to an Aachen author when surname and first name match exactly one author.
Applicants
- ACADEMISCH ZIEKENHUIS LEIDEN (H.O.D.N. LUMC) GBS Leiden
- アカデミス・ジーケンハイス・ライデン・ハー・オー・デー・エヌ・エルユーエムセー
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