By M. Ian Phillips (auth.), M. Ian Phillips PhD, DSc (eds.)

Announcement of the entire sequencing of the human genome in April 2003 validated the presence of millions of pursuits for antisense oligonucleotides and opened how you can 1000's of preclinical animal experiences and a few 20 ongoing scientific trials. during this moment version of Antisense Therapeutics, a workforce of best researchers and scientific scientists reveal the recent truth of antisense and RNA inhibition for treating a large diversity of ailments. The authors express how antisense oligonucleotides are being designed and studied when it comes to high blood pressure, numerous cancers, inflammatory bowel ailment, mind issues, the blood-brain barrier, and drug supply. Highlights contain RNA-based treatments for plenty of ailments, updated tools and purposes, and perception into the large power to supply a brand new iteration of gear.
hugely sensible and affliction orientated, Antisense Therapeutics, moment version bargains not just a primer for a brand new new release of drug discovery researchers, but additionally an illuminating exploration of the aptitude in exploiting RNA inhibition for novel human therapeutics.

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It was found that the status of the 5' hydroxyl terminus of the antisense strand of an siRNA determines RNAi activity, while a 3' terminus block is tolerated (56–58). Sequence mutations, on the other hand, are generally tolerated at the 5' end, but not the 3' end (59). Chemical modifications such as phosphorothioation and 2'-O-methylation and 2'-O-allylation were developed to improve the nuclease resistance of synthetic siRNAs (56,59,60). Certain chemical modifications at selected sites prolonged the siRNA activities, whereas others compromised the efficiency.

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48 Stark, G. , Kerr, I. , Williams, B. , et al. (1998) How cells respond to interferons. Annu. Rev. Biochem. 67, 227–264. Antisense Inhibition 31 49. Minks, M. , West, D. , and Baglioni, C. (1979) Structural requirements of double-stranded RNA for the activation of 2',5'-oligo(A) polymerase and protein kinase of interferon-treated Hela cells. J. Biol. Chem. 254, 10,180– 10,183. 50. 50 Elbashir, S. , et al. (2001) Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells.

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