Lentivirus has been shown to be an effective gene delivery tool and this, makes it a suitable gene therapy vector. Lentivirus vectors have been utilized for more than 236 clinical trials for gene delivery [4], and encouraging results in gene therapy that used recombinant lentivirus have been found from clinical trials for many diseases especially for cancer (Table 1). Due to the ability of down-regulating or up-regulating gene expression in vitro systems or animal models using lentiviral vector, researchers have employed these systems at high-throughput levels in order to determine the necessity and effects of transgenes in disease model systems and this is relevant in the discovery of new transgenic drugs. So far, the gene delivery into CD34+ HSCs using lentiviral vector has been applied in clinical trials and has been shown to be therapeutic in several diseases including; β-thalassemia [6], X-linked adrenoleukodystrophy (ALD) [7], metachromatic leukodystrophy [8,9], and Wiskott-Aldrich Syndrome [10]. Of them, HSCs transduced with β-globin in 5 patients with β-thalassemia one patient with βE/β0-thalassemia became weaned off transfusion [6]; HSCs transduced with wild-type ABCD1 in two patients with X-linked adrenoleukodystrophy (ALD), they obtained polyclonal reconstitution with 9 to 14% of granulocytes, monocytes, and T and B lymphocytes expressing the ALD protein with the stop of progressive cerebral demyelination [7]; The use of lentiviral vector to deliver functional WASP into three WAS patients to correct HSPCs’ genetic defect led to improvement in platelet count, immune function, and clinical state of the patients [10]. No side effects attributable to the vector have been reported in these Phase I/II clinical trials.

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