Drug Discovery Perspectives of Antisense Oligonucleotides
Biomolecules & Therapeutics, 2023 The era of innovative RNA therapies using antisense oligonucleotides (ASOs), siRNAs, and mRNAs is beginning. Since the emergence of the concept of ASOs in 1978, it took more than 20 years before they were developed into drugs for commercial use. Nine ASO drugs have been approved to date.
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Peripheral reduction of FGFR4 with antisense oligonucleotides increases metabolic rate and lowers adiposity in diet-induced obese mice. [PDF]
PLoS ONE, 2013 Obesity is a primary risk factor for multiple metabolic disorders. Many drugs for the treatment of obesity, which mainly act through CNS as appetite suppressants, have failed during development or been removed from the market due to unacceptable adverse ...
Xing Xian Yu +6 more
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Recent advances in malaria genomics and epigenomics [PDF]
, 2016 Malaria continues to impose a significant disease burden on low- and middle-income countries in the tropics. However, revolutionary progress over the last 3 years in nucleic acid sequencing, reverse genetics, and post-genome analyses has generated step ...
Kirchner, Sebastian +2 more
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PARP inhibitor ABT-888 affects response of MDA-MB-231 cells to doxorubicin treatment, targeting Snail expression [PDF]
, 2015 To overcome cancer cells resistance to pharmacological therapy, the development of new therapeutic approaches becomes urgent. For this purpose, the use of poly(ADP-ribose) polymerase (PARP) inhibitors in combination with other cytotoxic agents could ...
CAIAFA, Paola +11 more
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LIPID METABOLISM CORRECTION BY ANTISENSE TECHNOLOGY
Рациональная фармакотерапия в кардиологии, 2015 Antisense oligonucleotides (ASO) technology in elaboration of drugs for lipid metabolism correction is discussed. The main ASO types and modes of its action on the target mRNA are analyzed.
O. I. Afanasieva, S. N. Pokrovsky
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Dyclonine rescues frataxin deficiency in animal models and buccal cells of patients with Friedreich's ataxia. [PDF]
, 2014 Inherited deficiency in the mitochondrial protein frataxin (FXN) causes the rare disease Friedreich's ataxia (FA), for which there is no successful treatment. We identified a redox deficiency in FA cells and used this to model the disease.
Al-Mahdawi +78 more
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Antisense pro-drugs: 5'-ester oligodeoxynucleotides
Nucleic Acids Research, 1994 Oligonucleotides bearing a terminal lipophilic group attached through a biodegradable ester bond should be useful as antisense pro-drugs with improved cellular uptake. The synthesis of 5'-ester oligonucleotides is, however, problematic due to lability of the ester bond during aqueous ammonia treatment that is commonly used for the deprotection of ...
N N, Polushin, J S, Cohen
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Inhibition of Leishmania major PTR1 Gene Expression by Anti-sense in Escherichia coli [PDF]
Iranian Journal of Public Health, 2012 Background: Protozoa related to Trypanosome family including Leishmania, synthesize enzymes to escape from drug therapy. One of them is PTR1 that its enzymatic activity is similar to dihydrofolate reductase (DHFR).
F Kheirandish +5 more
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Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models. [PDF]
, 2019 Systemic delivery of antisense oligonucleotides (AO) for DMD exon skipping has proven effective for reframing DMD mRNA, rescuing dystrophin expression, and slowing disease progression in animal models.
Barthélémy, Florian +6 more
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Chronic myelogenous leukemia on target
Cancer Medicine, 2018 Chronic myelogenous leukemia (CML) is commonly treated with tyrosine kinase inhibitors (TKIs) that inhibit the pro‐leukemic activity of the BCR‐ABL1 oncoprotein.
Veronika Némethová, Filip Rázga
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