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Oligonucleotide-Based Drugs and Therapeutics - Nicolay

2018;84(1):64-77. 25. Niu C, Prakash TP, Kim A, et al. Antisense oligonucleotides targeting mutant ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7. Supportive Oligonucleotide Technique (SOT) is a treatment for cancer or infections with viruses or Lyme bacteria. Essentially SOT is the creation of a shutoff “key” that precisely fits a chosen “lock” portion of a cancer cell or pathogen.

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building blocks to our current work to expand the use of Oligonucleotide Therapeutics in the treatment of diseases across therapy areas within AstraZeneca. Postdoc in microbial biotechnology - wastewater treatment. Chalmers4,2 Research Scientist NGS/Transcriptomics – Oligonucleotides. AstraZeneca4,1.

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Oligonucleotide therapeutics are drugs that manipulate gene expression and improve the disease state. Antisense oligonucleotides hybridize with a target mRNA to downregulate gene expres … Alterations in pre-mRNA splicing can have profound effects on gene expression and lead to cellular transformation. This review discusses the six oligonucleotide therapeutic agents that have been FDA approved as of January 2017. They are fomivirsen (Vitravene), indicated for cytomegalovirus retinitis in HIV patients, a condition that no longer exists; pegaptanib (Macugen) indicated for wet macular degeneration of the retina; mipomersen (Kynamro), indicated for familial hypercholesterolemia; eteplirsen 2019-08-01 Antisense oligonucleotides represent a novel therapeutic platform for the discovery of medicines that have the potential to treat most neurodegenerative diseases.

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Aartsma-Rus A 1, Corey DR 2. Author information.

At one point, many pharmaceutical companies were conducting research on oligonucleotide therapy, which employs chemically synthesized nucleotide-like small molecule drugs with potential specificity similar to that of therapeutic antibodies. 2006-08-01 · A 20-mer oligonucleotide (SOD r146192) targeting superoxide dismutase 1 (SOD1) with phosphorothioate modifications throughout and 2′- O - (2-methoxy)ethyl substitutions on the sugars of the first and last 5 nucleotides to increase biological half-lives and binding affinity (refs. 13 – 15; see Methods) was continuously pumped into the right lateral ventricle of a normal rat via a catheter surgically implanted through the skull and connected to an osmotic pump imbedded subcutaneously. Mechanism of action of antisense therapy:
9. Translational arrest by blocking ribosome.
10. Activation of RNase enzyme:
11. Triplex antisense technology (ANTIGENE)
12.
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These agents primarily act by gene silencing or RNA interference. The most common side effect of oligonucleotide therapy is thrombocytopenia, a dangerously low platelet count that increases the risk of bleeding. The use of synthetic oligonucleotides in gene therapy is to inactivate the genes involved in the disease process. There are several methods by which this is achieved.

Oligonucleotides (ONs), the focus of the chapter herein, are mostly known for their utility to selectively manipulate RNA processing by increasing or decreasing target gene levels, in particular by inducing enzymatic RNA degradation, blocking or mimicking miRNAs, inhibiting mRNA translation or modulating pre-mRNA splicing. Mipomersen is an antisense oligonucleotide injectable drug that was recently approved by the Food and Drug Administration for the treatment of homozygous FH. It is complementary in sequence to a segment of the human apolipoprotein (Apo) B-100 messenger RNA and specifically binds to it, blocking translation of the gene product.
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Mechanism of action of antisense therapy:
9. Translational arrest by blocking ribosome.
10.

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Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice.

Proven expertise in oligonucleotide in vitro pharmacology* Representational Oligonucleotide Microarray Analysis (ROMA) detects genomic especially regarding the use or not of systemic therapies (hormonal therapy,  av U De Giorgi · 2005 · Citerat av 67 — Moreover, imatinib treatment in GIST represents an extraordinary model to an antisense oligonucleotide to the bcl-2 mRNA; Bevacizumab,  Research Scientist NGS/Transcriptomics – Oligonucleotides Do you want to explore and develop the science of oligonucleotide treatment, and have a passion  Are you an engaged scientist with molecular biology experience? Do you want to explore and develop the science of oligonucleotide treatment, and have a  av H Schmitt · 2020 · Citerat av 7 — agonist cobitolimod is a first-in-class DNA-based oligonucleotide DNA-based oligonucleotide with demonstrated therapeutic efficacy in  av X Huang · 2018 · Citerat av 30 — Importantly, Lu et al. showed that salinomycin treatment inhibits the Stetsenko, D. A.; Pyshnyi, D. V. Oligonucleotide Functionalization by a  I en fas 3-studie uppnåddes mycket goda resultat och preparatet fick i november »genombrottsstatus« (breakthrough therapy designation) av  Programmable Oligonucleotide Delivery System for next-generation siRNA Prostate cancer thErapy. Research project from 2020-10-01 to 2022-07-31.