Gene Therapy for Primary Immunodeficiency.
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ABSTRACT:
SUBMITTER: Houghton BC
PROVIDER: S-EPMC7773329 | biostudies-literature | 2021 Jan
REPOSITORIES: biostudies-literature
Publications
Gene Therapy for Primary Immunodeficiency.
Houghton Benjamin C BC Booth Claire C
HemaSphere 20201229 1
Over the past 3 decades, there has been significant progress in refining gene therapy technologies and procedures. Transduction of hematopoietic stem cells ex vivo using lentiviral vectors can now create a highly effective therapeutic product, capable of reconstituting many different immune system dysfunctions when reinfused into patients. Here, we review the key developments in the gene therapy landscape for primary immune deficiency, from an experimental therapy where clinical efficacy was mar ...[more]
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