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Antisense Oligonucleotide Therapies: Are They the “Magic Bullets”?

David D. F. Ma, MBBS, MD; and Trung Le Doan, PhD
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The Royal North Shore Hospital, Sydney, NSW, Australia 2065. Museum National d'Histoire Naturelle, 75231 Paris, France. Requests for Reprints: David D. F. Ma, MBBS, MD, Department of Haematology, The Royal North Shore Hospital, Sydney, NSW, Australia 2065.

Copyright ©2004 by the American College of Physicians

Ann Intern Med. 1994;120(2):161-163. doi:10.7326/0003-4819-120-2-199401150-00010
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The recent commencement of clinical trials using antisense oligonucleotide therapy for leukemias and viral infections has heralded a new era in drug therapy [14]. Antisense oligonucleotides are short chains of nucleic acids, usually 10 to 30 residues long, and are intermediate in size compared with smaller-size conventional drugs, such as β-blockers, and the much larger therapeutic polypeptides, such as growth factors or monoclonal antibodies. Conventional drugs generally affect cellular functions by interacting with proteins.

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