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
Bloodjournal_title
Bloodauthors
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 JMdoi
10.1182/blood-2006-11-058933subject
Has Abstractpub_date
2007-07-01 00:00:00pages
67-73issue
1eissn
0006-4971issn
1528-0020pii
blood-2006-11-058933journal_volume
110pub_type
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