Results 101 to 110 of about 270,275 (243)

Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders

University of Ottawa Journal of Medicine, 2018
Antisense oligonucleotides (ASOs) are synthetic, single-stranded DNA molecules that can bind to specific mRNA sequences and alter protein expression.
Ryan Gotesman
doaj   +1 more source

Oligonucleotide N3'-->P5' phosphoramidates as antisense agents. [PDF]

, 1996
Uniformly modified oligonucleotide N3'-->P5' phosphoramidates, where every 3'-oxygen is replaced by a 3'-amino group, were synthesized. These compounds have very high affinity to single-stranded RNAs and thus have potential utility as antisense agents.
CALABRETTA, Bruno, Chiu CY, Gryaznov S, Koziolkiewicz M, Nieborowska Skorska M, Lloyd D, Chen JK, Skorski T, Cucco C   +8 more
core  

Smad7 antisense oligonucleotide-based therapy for inflammatory bowel diseases

, 2013
The aetiology of both Crohn's disease and ulcerative colitis, the major forms of inflammatory bowel diseases in human beings, remains unknown. However, compelling evidence suggests that the associated pathological process in inflammatory bowel disease is
PALLONE, FRANCESCO, MONTELEONE, GIOVANNI, Monteleone, G, Sedda, S, Pallone, F, Zorzi, F, Angelucci, E   +6 more
core   +1 more source

Retro-1-Oligonucleotide Conjugates. Synthesis and Biological Evaluation

Molecules, 2019
Addition of small molecule Retro-1 has been described to enhance antisense and splice switching oligonucleotides. With the aim of assessing the effect of covalently linking Retro-1 to the biologically active oligonucleotide, three different derivatives ...
Jordi Agramunt, Enrique Pedroso, Silvia M. Kreda, Rudolph L. Juliano, Anna Grandas   +4 more
doaj   +1 more source

AZD9150, a Next-Generation Antisense Oligonucleotide Inhibitor of STAT3 with Early Evidence of Clinical Activity in Lymphoma and Lung Cancer

Science Translational Medicine, 2015
Systemically administered antisense oligonucleotide AZD9150 inhibits STAT3 and shows anticancer activity in preclinical models and patients. Blocking transcription in tumors, STAT STAT3 is a transcription factor that plays an oncogenic role in many ...
D. Hong, R. Kurzrock, Youngsoo Kim, R. Woessner, A. Younes, J. Nemunaitis, N. Fowler, Tianyuan Zhou, Joanna Schmidt, Minji Jo, Samantha J. Lee, Mason Yamashita, S. G. Hughes, L. Fayad, S. Piha-Paul, M. V. Nadella, Morvarid Mohseni, D. Lawson, C. Reimer, D. Blakey, Xiaokun Xiao, J. Hsu, A. Revenko, B. Monia, A. Macleod   +24 more
semanticscholar   +1 more source

Oligonucleotide Mimics for Antisense Therapeutics:  Solution Phase and Automated Solid-Support Synthesis of MMI Linked Oligomers

, 2016
Oligonucleotide Mimics for Antisense Therapeutics:  Solution Phase and Automated Solid-Support Synthesis of MMI Linked ...
Michel Perbost (2995368), Yogesh S. Sanghvi (2082307), Laurent Bellon (2995371), Jean-Jacques Vasseur (307813), François Morvan (2460325)   +4 more
core   +2 more sources

Local dystrophin restoration with antisense oligonucleotide PRO051

, 2007
Background: Duchenne's muscular dystrophy is associated with severe, progressive muscle weakness and typically leads to death between the ages of 20 and 35 years.
Goemans, Nathalie M., Janson, Anneke A., den Dunnen, Johan T., Verschuuren, Jan J., Ginjaar, Ieke B., Ekhart, Peter F., van Deutekom, Judith C., Aartsma-Rus, Annemieke, Koop, Klaas, Bremmer-Bout, Mattie, Frankhuizen, Wendy S., de Kimpe, Sjef J., van der Kooi, Anneke J., van Ommen, Gert-Jan B., Venneker, Edna H., Platenburg, Gerard J.   +15 more
core   +1 more source

Clinical and Preclinical Pharmacokinetics and Pharmacodynamics of Mipomersen (Kynamro®): A Second-Generation Antisense Oligonucleotide Inhibitor of Apolipoprotein B

Clinical Pharmacokinetics, 2015
Mipomersen (Kynamro®), a second-generation 2′-O-methoxyethyl chimeric antisense oligonucleotide (ASO), inhibits the synthesis of apolipoprotein B (apoB) and is indicated in the US as an adjunct therapy for homozygous familial hypercholesterolemia (HoFH ...
R. Geary, B. Baker, S. Crooke
semanticscholar   +1 more source

Gas-phase fragmentation and conformation of the sugar-modified antisense oligonucleotide tcDNA

, 2015
Tricyclo-DNA (tcDNA) is a sugar- and backbone-modified analogue of DNA that is currently tested as antisense oligonucleotide for the treatment of Duchenne muscular dystrophy.
Hari, Yvonne, Schürch, Stefan
core  

Cationic liposome-encapsulated antisense oligonucleotide mediates efficient killing of intracellular Leishmania

, 1999
Antisense oligonucleotides have been considered as inhibitors of growth of intracellular parasites such as Leishmania, but only limited inhibition has been observed in itro.
Dasgupta, Dalia, Chakraborty, Rila, Adhya, Samit, Chakroborty, Rila, Basu, Mukul K, Basu, Mukul K.   +5 more
core   +1 more source