Results 11 to 20 of about 270,275 (243)

Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects [PDF]

Molecular Therapy: Nucleic Acids
Nicotinamide N-methyltransferase (NNMT) is one of the methyltransferase family genes. It consumes S-adenosyl-l-methionine (SAM), which is required for DNA methylation and histone methylation for epigenetic regulation, to produce 1-methylnicotinamide from
Tomoaki Hara, Sikun Meng, Yuuya Kasahara, Takashi Osawa, Daisuke Motooka, Hiromichi Sato, Yasuko Arao, Yoshiko Saito, Kana Inoue, Yumiko Hamano, Yuichiro Doki, Hidetoshi Eguchi, Satoshi Obika, Hideshi Ishii   +13 more
doaj   +3 more sources

Pharmacokinetics of antisense oligonucleotide drugs

Фармакокинетика и Фармакодинамика, 2013
This review covers the pharmacokinetic characteristics of the various antisense oligonucleotide drugs. A comparison of the pharmacokinetics of drugs first and second generation.
M. R. Khaitov, V. V. Smirnov
doaj   +2 more sources

Antisense oligonucleotides [PDF]

Neurology Genetics, 2019
There are few disease-modifying therapeutics for neurodegenerative diseases, but successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy and Duchenne muscular dystrophy predict a robust future for ASOs in medicine.
Scoles, Daniel R., Minikel, Eric V., Pulst, Stefan M.   +2 more
openaire   +3 more sources

Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.

New England Journal of Medicine, 2022
BACKGROUND The intrathecally administered antisense oligonucleotide tofersen reduces synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with amyotrophic lateral sclerosis (ALS) associated with mutations in SOD1 (SOD1 ...
Timothy A. Miller, M. Cudkowicz, A. Genge, P. Shaw, G. Sobue, R. Bucelli, A. Chiò, P. van Damme, A. Ludolph, J. Glass, Jinsy A. Andrews, Suma Babu, M. Benatar, C. Mcdermott, T. Cochrane, Sowmya Chary, Sheena Chew, Handong Zhu, Fan Wu, I. Nestorov, D. Graham, P. Sun, M. McNeill, L. Fanning, T. Ferguson, S. Fradette   +25 more
semanticscholar   +1 more source

Antisense oligonucleotides for neurodegeneration [PDF]

Science, 2020
Promising clinical results for Huntington's disease give hope for other ...
Leavitt, BR, Tabrizi, SJ
openaire   +3 more sources

A perspective on oligonucleotide therapy: Approaches to patient customization

Frontiers in Pharmacology, 2022
It is estimated that the human genome encodes 15% of proteins that are considered to be disease-modifying. Only 2% of these proteins possess a druggable site that the approved clinical candidates target. Due to this disparity, there is an immense need to
Shikha Thakur, Apurba Sinhari, Priti Jain, Hemant R. Jadhav   +3 more
doaj   +1 more source

Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer’s disease: a phase 1b, randomized, placebo-controlled trial

Nature Medicine, 2023
Tau plays a key role in Alzheimer’s disease (AD) pathophysiology, and accumulating evidence suggests that lowering tau may reduce this pathology. We sought to inhibit MAPT expression with a tau-targeting antisense oligonucleotide (MAPT_Rx) and reduce tau
C. Mummery, A. Börjesson-Hanson, D. Blackburn, E. Vijverberg, P. D. de Deyn, S. Ducharme, Michael A G Jonsson, A. Schneider, J. Rinne, A. Ludolph, R. Bodenschatz, H. Kordasiewicz, E. Swayze, B. Fitzsimmons, L. Mignon, K. Moore, Chris Yun, Tiffany L. Baumann, Dan Li, D. Norris, R. Crean, Danielle L. Graham, E. Huang, E. Ratti, C. Bennett, Candice E. Junge, R. Lane   +26 more
semanticscholar   +1 more source

Neurofilament light‐chain response during therapy with antisense oligonucleotide tofersen in SOD1‐related ALS: Treatment experience in clinical practice

Muscle and Nerve, 2023
In amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 (SOD1) gene mutations (SOD1‐ALS), the antisense oligonucleotide tofersen had been investigated in a phase III study (VALOR) and subsequently introduced in an expanded access program.
Thomas Meyer, Peggy Schumann, P. Weydt, S. Petri, Y. Koc, S. Spittel, S. Bernsen, R. Günther, Jochen H Weishaupt, Marie Dreger, Felix Kolzarek, D. Kettemann, J. Norden, M. Boentert, Maximilian Vidovic, C. Meisel, C. Münch, A. Maier, Péter Körtvélyessy   +18 more
semanticscholar   +1 more source

Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

Nature Medicine, 2022
Fused in sarcoma (FUS) is an RNA-binding protein that is genetically and pathologically associated with rare and aggressive forms of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
Vladislav A. Korobeynikov, Alex K Lyashchenko, Beatriz Blanco-Redondo, Paayman Jafar-nejad, N. Shneider   +4 more
semanticscholar   +1 more source

Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial

Nature Medicine, 2022
CEP290-associated Leber congenital amaurosis type 10 (LCA10) is a retinal disease resulting in childhood blindness. Sepofarsen is an RNA antisense oligonucleotide targeting the c.2991+1655A>G variant in the CEP290 gene to treat LCA10. In this open-label,
S. Russell, A. Drack, A. Cideciyan, S. Jacobson, B. Leroy, C. van Cauwenbergh, A. Ho, A. Dumitrescu, Ian C. Han, Mitchell Martin, W. Pfeifer, E. Sohn, Jean Walshire, Alexandra V. Garafalo, A. Krishnan, Christian A Powers, A. Sumaroka, A. J. Roman, Eva Vanhonsebrouck, Eltanara Jones, F. Nerinckx, J. de Zaeytijd, R. Collin, C. Hoyng, P. Adamson, M. Cheetham, Michael R. Schwartz, W. den Hollander, F. Asmus, G. Platenburg, D. Rodman, A. Girach   +31 more
semanticscholar   +1 more source