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J Clin Invest. 2005 Aug;115(8):2064-7.

A new direction for gene therapy: intrathymic T cell-specific lentiviral gene transfer.

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  • 1National Heart, Lung, and Blood Institute, NIH, Bethesda, Maryland 20892, USA.

Abstract

Reports of neoplasia related to insertional activation of protooncogenes by retroviral vectors have raised serious safety concerns in the field of gene therapy. Modification of current approaches is urgently required to minimize the deleterious consequences of insertional mutagenesis. In this issue of the JCI, Adjali and colleagues report on their treatment of SCID mice lacking the 70-kDa protein tyrosine kinase, ZAP-70, with direct intrathymic injection of a ZAP-70-expressing T cell-specific lentiviral vector, which resulted in T cell reconstitution. Using lentiviral vectors and in situ gene transfer may represent a safer approach than using retroviral vectors for ex vivo gene transfer into HSCs, avoiding 3 factors potentially linked to leukemogenesis, namely HSC targets, ex vivo transduction and expansion, and standard Moloney leukemia virus-based retroviral vectors.

PMID:
16075048
PMCID:
PMC1180563
DOI:
10.1172/JCI26041
[PubMed - indexed for MEDLINE]
Free PMC Article
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