Journal
DRUG DISCOVERY TODAY
Volume 13, Issue 11-12, Pages 513-521Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.drudis.2008.03.014
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Funding
- NCI NIH HHS [R01 CA112029, R01 CA121211-01A1, R01 CA112029-04, T32 CA075930, R01 CA121211, T32 (CA075930), R01 CA112029-03] Funding Source: Medline
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Antisense oligonucleotides can regulate gene expression in living cells. As such, they regulate cell function and division, and can modulate cellular responses to internal and external stresses and stimuli. Although encouraging results from preclinical and clinical studies have been obtained and significant progress has been made in developing these agents as drugs, they are not yet recognized as effective therapeutics. Several major hurdles remain to be overcome, including problems with efficacy, Off-target effects, delivery and side effects. The lessons learned from antisense drug development can help in the development of other oligonucleotide-based therapeutics such as CpG oligonucleotides, RNAi and miRNA.
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