Download Antisense Drug Technology: Principles, Strategies, and by Stanley T. Crooke PDF

By Stanley T. Crooke

Generally revised and up to date, Antisense Drug know-how: ideas, innovations, and functions, moment version displays the logarithmic development made long ago 4 years of oligonucleotide-based treatments, and, specifically, antisense therapeutics and study. reading classes realized from the scientific trials of first new release medications, the booklet evaluates the expertise as an entire and gives new instructions and avenues of study and improvement. Divided into 5 components, the publication starts off with a radical creation to the mechanism of antisense drug motion together with the RNase H mechanism, small RNA silencing pathways, and the capability therapeutics of splice switching oligonucleotides. top researchers show the fundamentals of oligonucleotide therapeutics partly by way of delineating medicinal chemistry, pharmacokinetics, and supply routes corresponding to liposomal formulations for nucleic acid delivery.  half 3 information hybridization established medicinal drugs and considers the dramatic advances represented through 2’ methoxyethyl chimeric antisense inhibitors and duplex RNA medicinal drugs. different chemical periods of gear and mechanisms of motion are defined partly 4 with extra discussions on bettering the second one new release antisense medicinal drugs. the ultimate half delves deeply into healing functions. Contributing authors study the opportunity of antisense medicinal drugs for the relief of cardiovascular ailments, metabolic illnesses, inflammatory ailments, melanoma, neurological issues, and immune modulation. featuring a hugely specified, lucid dialogue of the outstanding advances within the box, Antisense Drug expertise: rules, concepts, and purposes, moment variation offers the platform for researchers to proceed to aggressively pursue the nice chance represented by way of this interesting expertise.

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Additional resources for Antisense Drug Technology: Principles, Strategies, and Applications, Second Edition

Example text

So both the 5Ј and 3Ј splice sites are amenable to the effects of antisense drugs. Is there then a pattern of sensitivity with regard to the strength of the splicing signal? If there is, it is certainly neither obvious nor universal. One of the problems, of course, is deciding how strong a splice signal is. Using the classification of 5Ј splice sites proposed by Roca et al. qxd 5/24/2007 21:54 Page 23 MECHANISMS OF ANTISENSE DRUG ACTION, AN INTRODUCTION 23 Isis involving many targets. Although it is even more difficult to assign a “strength” value for 3Ј splice sites, certainly no pattern emerges [89].

Indeed, that has been our experience. For example, we have compared the activities of lower- and higher-affinity antisense drugs that work via an RNase H (RNase H enzymes are double-strand RNA-binding proteins that cleave RNA in an RNA–DNA duplex) mechanism against a number of cellular targets. In addition to the expected increase in potency, many more sites within the RNA were accessible to the higher-affinity class of antisense drugs for all the transcripts studied. 2 Specificity for Nucleic Acid Sequences Specificity derives from the selectivity of Watson–Crick or other types of base pairing.

Thus, a single base mismatch could result in a change in affinity of approximately 500-fold [53]. Modifications of oligonucleotides may alter specificity. At the genomic level, any sequence of 17 residues is expected to occur only once [54]. Assuming a random distribution of sequences in RNA, any sequence of 13 residues is expected to occur once in the cellular RNA population and, if the nonrandom nature of mammalian RNA sequence is taken into account, an 11-mer or perhaps smaller oligonucleotide could identify and bind to a unique sequence [55].

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