Results 111 to 120 of about 266,614 (380)

Antisense-mediated splice intervention to treat human disease: the odyssey continues [version 1; peer review: 3 approved]

F1000Research, 2019
Recent approvals of oligonucleotide analogue drugs to alter gene expression have been welcomed by patient communities but not universally supported. These compounds represent a class of drugs that are designed to target a specific gene transcript, and ...
Ianthe Pitout, Loren L. Flynn, Steve D. Wilton, Sue Fletcher   +3 more
doaj   +1 more source

Antisense oligonucleotide-mediated MDM4 exon 6 skipping impairs tumor growth.

Journal of Clinical Investigation, 2016
MDM4 is a promising target for cancer therapy, as it is undetectable in most normal adult tissues but often upregulated in cancer cells to dampen p53 tumor-suppressor function.
M. Dewaele, Tommaso Tabaglio, K. Willekens, M. Bezzi, S. Teo, Diana H. P. Low, Cheryl M. Koh, F. Rambow, M. Fiers, A. Rogiers, E. Radaelli, M. Al-Haddawi, Soo-Yong Tan, Els Hermans, F. Amant, Hualong Yan, Manikandan Lakshmanan, Ratnacaram Chandrahaas Koumar, S. Lim, Frederick A. Derheimer, R. Campbell, Zahid Bonday, V. Tergaonkar, M. Shackleton, C. Blattner, J. Marine, E. Guccione   +26 more
semanticscholar   +1 more source

Exome Sequencing Reveals the Genetic Architecture of Non‐syndromic Orofacial Clefts and Identifies BOC as a Novel Causal Gene

Advanced Science, EarlyView.
Nonsyndromic orofacial clefts (NSOFCs) are the most common craniofacial defects. Exome sequencing of 214 sporadic cases sheds new light on its genetic architecture and identifies many candidate pathogenic variants. Furthermore, functional studies establish BOC as a novel causal gene and reveal an unusual two‐locus model of inheritance via the epistatic
Qing He, Min Yu, Yuhua Jiao, Yizhu Xu, Xuqin Liang, Wenbin Huang, Linping Xu, Yuxia Hou, Zhanping Ren, Beile Lyu, Zhenwei Qian, Pengpeng Liu, Jing Zhou, Huimei Huang, Chunyan Yin, Huaxiang Zhao, Yi Ding   +16 more
wiley   +1 more source

ISS-N1 makes the first FDA-approved drug for spinal muscular atrophy

Translational Neuroscience, 2017
Spinal muscular atrophy (SMA) is one of the leading genetic diseases of children and infants. SMA is caused by deletions or mutations of Survival Motor Neuron 1 (SMN1) gene.
Ottesen Eric W.
doaj   +1 more source

Fluorescent labeling of plasmid DNA and mRNA : gains and losses of current labeling strategies [PDF]

, 2016
Live-cell imaging has provided the life sciences with insights into the cell biology and dynamics. Fluorescent labeling of target molecules proves to be indispensable in this regard. In this Review, we focus on the current fluorescent labeling strategies
Rombouts, Koen, Braeckmans, Kevin, Remaut, Katrien   +2 more
core   +1 more source

Tissue pharmacokinetics of antisense oligonucleotides

Molecular Therapy - Nucleic Acids
Pharmacokinetics (PK) of antisense oligonucleotides (ASOs) is characterized by rapid distribution from plasma to tissue and slow terminal plasma elimination driven by re-distribution from tissue. Quantitative understanding of tissue PK and RNA knockdown for various ASO chemistries, conjugations, and administration routes is critical for successful drug
Erica Bäckström, Alessandro Bonetti, Per Johnsson, Stefan Öhlin, Anders Dahlén, Patrik Andersson, Shalini Andersson, Peter Gennemark   +7 more
openaire   +3 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   +2 more sources

Epigenetic Activation of CCDC183‐AS1 Promotes Osteoclastogenesis and Prostate Cancer Bone Metastasis Through the FUBP1/LIGHT Axis

Advanced Science, EarlyView.
CCDC183‐AS1 overexpression enhanced the ability of PCa cells to spread to the bone by inducing osteoclastogenesis. Mechanistically, CCDC183‐AS1 interacted with FUBP1 and enhanced its stability, which promoted the transcription of TNFSF14 (LIGHT). Copy number gain‐induced KDM5C epigenetically upregulated CCDC183‐AS1 expression by recruiting TET1 to the ...
Chuandong Lang, Xiangyu Mu, Kun Chen, Xinwen Wang, Yuluo Rong, Jia Wang, Zongcheng Yang, Chi Yin, Yuhu Dai, Jun Xiao, Wenzhi Zhang   +10 more
wiley   +1 more source

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  

Antisense oligonucleotide based therapeutics and its applications against bacterial infections

Medicine in Drug Discovery, 2023
The transcriptome of a cell governs the overall cellular expression of proteins, hence, targeting mRNA holds promise as a therapeutic strategy in the field of drug discovery.
Nupur Angrish, Garima Khare
doaj