Since the commencement of the first clinical trial involving gene transfer into human subjects in 1989, more than 100 patients have been treated and more than 100 patients have been treated and more than 70 clinical trials are underway.
Many of the ongoing clinical trials are gene marking trials, in which genes are introduced into cells prior to transplantation to study their fate and function. The majority of current clinical trials are aimed at gene therapy for cancer.
Other are aimed at exploring the potential of gene therapy for inborn errors of metabolism including adenosine deaminase deficiency,
familial hypercholesterolemia, Gaucher’s disease, and cystic fibrosis.
The first therapeutic trials involved the introduction of a gene for adenosine deaminase into the cultured lymphocytes from children with severe combined immunodeficiency secondary to adenosine deaminase deficiency. These cells were subsequently transfused into patients to provide essential cultured hepatocytes from patients with familial hypercholesterolemia and subsequent transplantation of these genetically reconstitude cells into the liver. Both of these trials employed retroviral vectors. More recently, trials for cystic fibrosis have begun using several adenoviral as well as DNA vectors instilled directly into the trachea to achieve expression of the CFTR gene in the lung. Several centers have also proposed therapy for Gaucher’s disease using an ex vivo procedure in which bone marrow is harvested from patients with severe forms of the disorder, infected with retroviral vectors to replace the defective gene, and then transplanted back into the patient.
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