Document 0551 DOCN M9460551 TI The new frontier: gene and oligonucleotide therapy. DT 9404 AU Schreier H; Center for Lung Research, Vanderbilt University School of; Medicine, Nashville, TN 37232-2650. SO Pharm Acta Helv. 1994 Jan;68(3):145-59. Unique Identifier : AIDSLINE MED/94167392 AB 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. DE Animal *Gene Therapy Human *Oligonucleotides Support, Non-U.S. Gov't JOURNAL ARTICLE REVIEW REVIEW, TUTORIAL SOURCE: National Library of Medicine. NOTICE: This material may be protected by Copyright Law (Title 17, U.S.Code).