Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency.
Blood
; 110(1): 67-73, 2007 Jul 01.
Article
em En
| MEDLINE
| ID: mdl-17369490
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.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
/
Receptores de Interleucina-2
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Doenças por Imunodeficiência Combinada Ligada ao Cromossomo X
/
Imunidade
Limite:
Humans
/
Infant
Idioma:
En
Ano de publicação:
2007
Tipo de documento:
Article