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Gene therapy for primary immunodeficiency.
Gene therapy for primary immunodeficiency. Human molecular genetics Booth, C., Romano, R., Roncarolo, M. G., Thrasher, A. J. 2019Abstract
Gene therapy is now being trialled as a therapeutic option for an expanding number of conditions, based primarily on the successful treatment over the past two decades of patients with specific primary immunodeficiencies (PIDs) including severe combined immunodeficiency (SCID) and Wiskott-Aldrich syndrome (WAS) and metabolic conditions such as leukodystrophy. The field has evolved from the use of gammaretroviral vectors to more sophisticated lentiviral platforms which offer an improved biosafety profile alongside greater efficiency for HSC gene transfer. Here we review more recent developments including licensing of gene therapies, use of gene corrected autologous T cells as an alternative strategy for some PIDs and the potential of targeted gene correction using various gene editing platforms. Given the promising results of recent clinical trials it is likely that autologous gene therapies will become standard of care for a number of devastating diseases in the coming decade.
View details for DOI 10.1093/hmg/ddz170
View details for PubMedID 31297531