By M. Ian Phillips (auth.), M. Ian Phillips PhD, DSc (eds.)
Announcement of the full sequencing of the human genome in April 2003 proven the presence of millions of ambitions for antisense oligonucleotides and opened find out how to 1000s of preclinical animal stories and a few 20 ongoing medical trials. during this moment version of Antisense Therapeutics, a staff of best researchers and medical scientists show the recent truth of antisense and RNA inhibition for treating a extensive diversity of ailments. The authors express how antisense oligonucleotides are being designed and studied with regards to high blood pressure, numerous cancers, inflammatory bowel sickness, mind issues, the blood-brain barrier, and drug supply. Highlights comprise RNA-based remedies for plenty of ailments, up to date tools and purposes, and perception into the big capability to supply a brand new iteration of substances.
hugely useful and sickness orientated, Antisense Therapeutics, moment variation bargains not just a primer for a brand new new release of drug discovery researchers, but in addition an illuminating exploration of the aptitude in exploiting RNA inhibition for novel human therapeutics.
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Extra info for Antisense Therapeutics
1. PGMC Delivery Assisted by Diagnostic Ultrasound Based on the aforementioned advantages, 21 pigs received AVI-4217 (a pig version of AVI-4126) bound to PGMC, AVI-4217 alone, or no antisense treatment after carotid balloon injury (25). The vessels were evaluated 30 d postinjury. The results are presented in Table 7. 2. PGMC for Site-Specific Delivery of AVI-4217 in Porcine Coronary Vessels The aforementioned results are impressive, so additional studies that did not involve ultrasound for site-specific delivery of the AVI-4217 were conducted in coronary vessels with stents.
Work is currently being done to engineer DNAzymes, which should be more stable than their ribozyme RNA counterparts (33). One benefit of exogenous ribozyme delivery in vivo is that the immune system is fairly tolerant of foreign RNA molecules (34). The other method for delivering ribozymes, endogenous expression of the ribozyme, is most often accomplished using viral vectors; however, plasmid vectors may also be used. Both retroviral and DNA viral vectors have been used. Expression cassettes can be designed to carry cell type–specific or conditional transcription initiation sites, as well as to include reporter proteins.
10 Summerton, J. (1999) Morpholino antisense oligomers: the case for an RNase— independent structure type. Biochim. Biophys. Acta 1489, 141–158. 11. Elayadi, A. N. and Corey, D. R. (2001) Application of PNA and LNA oligomers to chemotherapy. Curr. Opin. Investig. Drugs 2, 558–561. 12. , Read, R. , and Van Meervelt, L. (2002) Crystal structure of double helical hexitol nucleic acids. J. Am. Chem. Soc. 124, 928–933. Antisense Inhibition 29 13. , and Zhang, R. (2002) Antisense anti-MDM2 mixed-backbone oligonucleotides enhance therapeutic efficacy of topoisomerase I inhibitor irinotecan in nude mice bearing human cancer xenografts: in vivo activity and mechanisms.
Antisense Therapeutics by M. Ian Phillips (auth.), M. Ian Phillips PhD, DSc (eds.)