The new frontier: gene and oligonucleotide therapy.
Gene and oligonucleotide therapy are emerging as clinically viable therapeutic regimens for genetic, neoplastic, and infectious diseases. Approaches include insertion of human genes in viral vectors including recombinant retrovirus, adenovirus, adeno-associated virus, and herpes simplex virus-1, or recombinant bacterial plasmids. Viral vectors transfect cells directly; plasmid DNA is delivered with the help of cationic liposomes (lipofection), polylysine conjugates, gramicidin S, artificial viral envelopes or other such intracellular carriers. Major areas of interest include replacement of the cystic fibrosis transmembrane regulator gene and the alpha 1-antitrypsin gene; arrest of human immunodeficiency virus infection; and reversal of tumorigenicity and cancer immunization, among others. Oligonucleotide therapy is principally focusing on the same areas, although the approach is to halt DNA transcription or messenger RNA translation with code-blocking triple-helix-forming or "antisense" oligomers. Contributions from the pharmaceutical sciences are expected in pharmaceutical chemistry, drug delivery systems design, analytical chemistry, and biopharmaceutics.
Schreier
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Center for Lung Research, Vanderbilt University School of Medicine, Nashville, TN 37232-2650.
D000818:Animals; D015316:Genetic Therapy; D006801:Humans; D009841:Oligonucleotides
D009841:Oligonucleotides