Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency.

Abstract:

:Retroviral gene therapy can restore immunity to infants with X-linked severe combined immunodeficiency (XSCID) caused by mutations in the IL2RG gene encoding the common gamma chain (gammac) of receptors for interleukins 2 (IL-2), -4, -7, -9, -15, and -21. We investigated the safety and efficacy of gene therapy as salvage treatment for older XSCID children with inadequate immune reconstitution despite prior bone marrow transplant from a parent. Subjects received retrovirus-transduced autologous peripherally mobilized CD34(+) hematopoietic cells. T-cell function significantly improved in the youngest subject (age 10 years), and multilineage retroviral marking occurred in all 3 children.

journal_name

Blood

journal_title

Blood

authors

Chinen J,Davis J,De Ravin SS,Hay BN,Hsu AP,Linton GF,Naumann N,Nomicos EY,Silvin C,Ulrick J,Whiting-Theobald NL,Malech HL,Puck JM

doi

10.1182/blood-2006-11-058933

subject

Has Abstract

pub_date

2007-07-01 00:00:00

pages

67-73

issue

1

eissn

0006-4971

issn

1528-0020

pii

blood-2006-11-058933

journal_volume

110

pub_type

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