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https://www.nature.com/articles/nrneurol.2017.148
Dec 01, 2017 · Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS. Nucleic Acids Res. 41 , …Author: C Rinaldi, Wood Mja.
https://www.sigmaaldrich.com/technical-documents/articles/biology/antisense-oligonucleotides.html
Delivery is key: lessons learnt from developing splice-switching antisense therapies. EMBO Mol Med, 9, 545-57. Sun Y, Zhao Y, Zhao X, Lee RJ, Teng L, & Zhou C (2017). Enhancing the Therapeutic Delivery of Oligonucleotides by Chemical Modification and Nanoparticle Encapsulation. Molecules, 22, pii: E1724. Krhac Levacic A, Morys S, & Wagner E (2017).
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2559958/
Design and Delivery of Antisense Oligonucleotides to Block microRNA Function in Cultured Drosophila and Human Cells Michael D. Horwich and Phillip D. Zamore Department of Biochemistry and Molecular Pharmacology and Howard Hughes Medical Institute, University of Massachusetts Medical School, Worcester, MA 01605Author: Michael D Horwich, Phillip D Zamore
https://www.ncbi.nlm.nih.gov/pubmed/9212909
Morpholino antisense oligomers: design, preparation, and properties. ... , most notably inadequate specificity, ineffective delivery into the proper subcellular compartment, and unpredictable activity within cells. Herein is an overview of the design, preparation, and properties of Morpholino oligos, a novel antisense structural type that ...Author: James Summerton, Dwight Weller
https://mct.aacrjournals.org/content/1/5/347
Mar 01, 2002 · The use of vectors in antisense drug delivery in vivo remains, at this point, a somewhat open question. In contrast to in vitro studies, all of the clinical trials with antisense oligonucleotides are carried out with naked oligonucleotides (51, 104). A delivery vehicle does not appear to be needed as endosomal/lysosomal sequestration, and lack ...Author: Nathalie Dias, C. A. Stein
https://www.bio-rad.com/en-us/applications-technologies/oligonucleotides-design-applications?ID=MOYTCO15
Antisense oligonucleotides are used to reduce levels of protein synthesis by inhibiting mRNA processing or translation. This approach is the basis of many therapies that are now in clinical trials including a range of cancer treatments. Antisense therapy is currently available for cytomegalovirus retinitis and familial hypercholesterolemia.
https://onlinelibrary.wiley.com/doi/full/10.1111/j.1440-1681.2006.04403.x
May 12, 2006 · ANTISENSE OLIGONUCLEOTIDES: FROM DESIGN TO THERAPEUTIC APPLICATION. Jasmine HP Chan. ... Modes of action of antisense oligonucleotides (ASOs). In the absence of ASO, normal gene and protein expression is maintained. ... It enhances ASO cellular delivery into the cytosol and nucleus and increases the retention time of ASO in the cells ...Author: Jasmine H. P. Chan, Shuhui Lim, W. S. Fred Wong
https://www.oligotherapeutics.org/antisense-oligonucleotides-mechanisms-of-action-and-rational-design/
Robert MacLeod. Dr. MacLeod is currently the Vice President Oncology Research and Development, and Franchise Head at Ionis Pharmaceuticals and has contributed to the discovery and development of clinical drug candidates in the several therapeutic areas including, oncology, thrombosis, inflammatory and muscle diseases.
https://advances.sciencemag.org/content/4/10/eaat3386.full
Oct 01, 2018 · Antisense oligonucleotide (ASO) silencing of the expression of disease-associated genes is an attractive novel therapeutic approach, but treatments are limited by the ability to deliver ASOs to cells and tissues. Following systemic administration, ASOs preferentially accumulate in liver and kidney. Among the cell types refractory to ASO uptake is the pancreatic insulin-secreting β-cell. Here ...Author: C. Ämmälä, W. J. Drury, L. Knerr, I. Ahlstedt, P. Stillemark-Billton, C. Wennberg-Huldt, E.-M. Ander...
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