Progress Toward Human Gene Therapy

Current aspects of gene therapy: implications for vascular interventions

“…A total of 52 gene transfer protocols had approved in the United States by December 1993, of which more than 43 involved gene therapeutic approaches [2,4,13,14]. Current strategies for gene therapy can be summarized as follows: The first class of experiments involves the transfer of genes to treat single-gene inherited disorders, for example, the gene defect in severe combined immune deficiency and that for the low-density lipoprotein receptor in familial hypercholesterolemia.…”

“…Ad5 is an icosahedral, nonenveloped virus carrying a linear, double-stranded 36 kb DNA genome [2,4,14,[21][22][23]. Ad5 is an icosahedral, nonenveloped virus carrying a linear, double-stranded 36 kb DNA genome [2,4,14,[21][22][23].…”

“…Ad5 is an icosahedral, nonenveloped virus carrying a linear, double-stranded 36 kb DNA genome [2,4,14,[21][22][23]. Generally 293 cells (human embryonic kidney cells) are used for this purpose [1,2,7,12,14,[21][22][23]. This makes the virus unable to replicate.…”

“…A major advantage of adenoviral vectors for gene therapy of cardiovascular disease is the successful infection of nonreplicating cells [1,2,4,6,7,14,16,[21][22][23]. Adenoviral vectors also accommodate larger cDNA inserts, up to 14 kb, than do retroviral vectors.…”

Gene therapy for PNET

“…The term 'gene therapy' refers specifically to the alteration of the genome of all or a portion of an organism's cells, leading to specific changes in the phenotype of the altered cells [1][2][3]. Gene therapy approaches for malignant neoplasms have been directed at both the host's cells and at the tumor cells.…”