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Article

Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

31 Mar 2020

Abstract excerpt

Recombinase-activating gene-1 (RAG1)-deficient severe combined immunodeficiency (SCID) patients lack B and T lymphocytes due to the inability to rearrange immunoglobulin and T cell receptor genes. Gene therapy is an alternative for those RAG1-SCID patients who lack a suitable bone marrow donor. We designed lentiviral vectors with different internal promoters driving codon-optimized RAG1 to ensure optimal...

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PMID: 32322605
DOI: 10.1016/j.omtm.2020.03.016

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