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Gene Therapies for Primary Immune Deficiencies

25 Feb 2021

Abstract excerpt

Gene therapy is an innovative treatment for Primary Immune Deficiencies (PIDs) that uses autologous hematopoietic stem cell transplantation to deliver stem cells with added or edited versions of the missing or malfunctioning gene that causes the PID. Initial studies of gene therapy for PIDs in the 1990-2000's used integrating murine gamma-retroviral vectors. While these studies showed clinical efficacy in many...

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PMID: 33717203
DOI: 10.3389/fimmu.2021.648951

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