Results 91 to 100 of about 127,002 (321)

Delivery is key: lessons learnt from developing splice‐switching antisense therapies

EMBO Molecular Medicine, 2017
The use of splice‐switching antisense therapy is highly promising, with a wealth of pre‐clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety of disorders has yet to be realized. The main obstacle impeding the
Caroline Godfrey, Lourdes R Desviat, Bård Smedsrød, France Piétri‐Rouxel, Michela A Denti, Petra Disterer, Stéphanie Lorain, Gisela Nogales‐Gadea, Valentina Sardone, Rayan Anwar, Samir EL Andaloussi, Taavi Lehto, Bernard Khoo, Camilla Brolin, Willeke MC van Roon‐Mom, Aurélie Goyenvalle, Annemieke Aartsma‐Rus, Virginia Arechavala‐Gomeza   +17 more
doaj   +1 more source

The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice

Molecular Therapy: Nucleic Acids, 2014
Antisense-mediated exon skipping is currently in clinical development for Duchenne muscular dystrophy (DMD) to amend the consequences of the underlying genetic defect and restore dystrophin expression. Due to turnover of compound, transcript, and protein,
Ingrid E C Verhaart, Laura van Vliet-van den Dool, Jessica A Sipkens, Sjef J de Kimpe, Ingrid G M Kolfschoten, Judith C T van Deutekom, Lia Liefaard, Jim E Ridings, Steve R Hood, Annemieke Aartsma-Rus   +9 more
doaj   +1 more source

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, Douine, Emilie D, Hsu, Christopher, Marcantonio, Eugene E, Miceli, M Carrie, Nelson, Stanley F, Wang, Richard T   +6 more
core   +1 more source

Nap1L4a Cooperates with Scl/Klf1 to Recruit H2A.Z in Mediating Interactions Among Cis‐Regulatory Elements and Transcription Required for Primitive Erythropoiesis in Zebrafish

Advanced Science, EarlyView.
Nap1l4a is required in erythropoiesis and hypoxia responses via physical interaction with Klf1 and Scl to recruit the histone variant H2A.Z. This facilitates its associated cis‐regulatory element (CRE) remodeling and the consequent chromatin assembly, and activates the transcription of erythroid lineage‐specific genes.
JiaHao Shi, FuMing Lai, Zheng Shen, XiaoYan Zhang, HanFei Wang, WenYe Liu, YunLong Wang, KuanYu Li, GuoLiang Li, YaPing Fang, Jing‐Xia Liu   +10 more
wiley   +1 more source

Transforming RNA Degradation into Accurate RNA Detection: RNase I–Assisted Rolling Circle Amplification for Clinical Samples

Angewandte Chemie, EarlyView.
Here, we transform this otherwise destructive enzymatic activity into a powerful diagnostic advantage through an RNase I–assisted rolling circle amplification (RI‐RCA) strategy. By integrating controlled RNase I–mediated RNA digestion with circular DNA templates, this approach enables direct and highly sensitive detection of target RNA sequences. Using
Amal Mathai, Cheng‐Yu Lee, Zijie Zhang, Jimmy Gu, John D. Brennan, Yingfu Li   +5 more
wiley   +2 more sources

ALS antisense oligonucleotides [PDF]

Science-Business eXchange, 2013
Isis and three academic groups are developing antisense oligonucleotide therapeutics for the most common cause of ALS. The drugs target hexanucleotide repeat expansions in C9orf72 and mitigate neurotoxicity in vitro. Animal models are not yet available.
openaire   +1 more source

OCTN2 Activates a Non‐Canonical Carnitine Metabolic Pathway to Promote MASH‐HCC Progression and Immunotherapy Resistance

Advanced Science, EarlyView.
In non‐MASH‐HCC, L‐carnitine promotes tumor progression primarily through its classical role in enhancing fatty acid oxidation (FAO). However, in MASH‐HCC, where FAO is markedly suppressed, L‐carnitine shifts from this canonical function to serve instead as an intracellular acetyl group buffer.
Chuqi Xia, Xiao Zhang, Jinze Li, Ning Xu, Sheng Hu, Qiyu Lu, Yuxuan Li, Taifu Xiao, Xu Li, Xue Wang, Kequan Xu, Daoming Liang   +11 more
wiley   +1 more source

Rethinking antisense oligonucleotide therapeutics for amyotrophic lateral sclerosis

Annals of Clinical and Translational Neurology
Antisense oligonucleotides, which are used to silence target genes, are gaining attention as a novel drug discovery modality for proteinopathies. However, while clinical trials for neurodegenerative diseases like amyotrophic lateral sclerosis have been ...
Daisuke Ito, Kensuke Okada
doaj   +1 more source

Antisense oligonucleotides in neurological disorders

Therapeutic Advances in Neurological Disorders, 2018
The introduction of genetics revolutionized the field of neurodegenerative and neuromuscular diseases and has provided considerable insight into the underlying pathomechanisms. Nevertheless, effective treatment options have been limited.
Claudia D. Wurster, Albert C. Ludolph
doaj   +1 more source

Evidence for regulated expression of Telomeric Repeat-containing RNAs (TERRA) in parasitic trypanosomatids [PDF]

, 2017
The Telomeric Repeat-containing RNAs (TERRA) participate in the homeostasis of telomeres in higher eukaryotes. Here, we investigated the expression of TERRA in Leishmania spp.
Damasceno, Jeziel D., Silva, Gabriel L.A., Tosi, Luiz R.O., Tschudi, Christian   +3 more
core   +1 more source