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Blood, Vol. 95 No. 8 (April 15), 2000:
pp. 2499-2504
REVIEW ARTICLE
Development of lentiviral vectors for gene therapy for human
diseases
Gary L. Buchschacher Jr and
Flossie Wong-Staal
From the Division of Hematology/Oncology and the Departments of
Medicine and Biology, University of California at San Diego, La Jolla,
CA.
Retroviral vectors derived from murine retroviruses are being used
in several clinical gene therapy trials. Recently, progress has been
made in the development of vectors based on the lentivirus genus of
retroviruses, which ironically includes a major human pathogen, human
immunodeficiency virus (HIV). As these vector systems for clinical gene
transfer are developed, it is important to understand the rationale
behind their design and development. This article reviews the
fundamental features of retrovirus replication and of the elements
necessary for development of a retroviral vector system, and it
discusses why vector systems based on HIV or other lentiviruses have
the potential to become important tools in clinical gene therapy.
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