Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

Cavazzana, Marina; Six, Emmanuelle; Lagresle‐Peyrou, Chantal; André‐Schmutz, Isabelle; Hacein‐Bey‐Abina, Salima

doi:10.1089/hum.2015.137

Public

Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

Published in Human Gene Therapy • Jan 20, 2016

Authors:

Marina Cavazzana

Emmanuelle Six

Chantal Lagresle‐Peyrou

Abstract

More than 20 years ago, X-linked severe combined immunodeficiency (SCID-X1) appeared to be the best condition to test the feasibility of hematopoietic stem cell gene therapy. The seminal SCID-X1 clinical studies, based on first-generation gammaretroviral vectors, demonstrated good long-term immune r...

Subject

Genetic enhancement

Severe combined immunodeficiency

Retrovirus

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