Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning

Aiuti, Alessandro; Slavin, Shimon; Aker, Memet; Ficara, Francesca; Deola, Sara; Mortellaro, Alessandra; Morecki, Shoshana; Andolfi, Grazia; Tabucchi, Antonella; Carlucci, Filippo; Marinello, E.; Cattaneo, Federica; Vai, Sergio; Servida, Paolo; Miniero, R; Grazia Roncarolo, Maria; Bordignon, Claudio

doi:10.1126/science.1070104

Public

Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning

Published in Science • Jun 28, 2002

Authors:

Alessandro Aiuti

Shimon Slavin

Memet Aker

Abstract

Hematopoietic stem cell (HSC) gene therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID) has shown limited clinical efficacy because of the small proportion of engrafted genetically corrected HSCs. We describe an improved protocol for gene transfer into HSCs associa...

Subject

Genetic enhancement

Severe combined immunodeficiency

Adenosine deaminase deficiency

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