Results 91 to 100 of about 275,477 (365)

Splice-switching antisense oligonucleotides as therapeutic drugs

Nucleic Acids Research, 2016
Splice-switching oligonucleotides (SSOs) are short, synthetic, antisense, modified nucleic acids that base-pair with a pre-mRNA and disrupt the normal splicing repertoire of the transcript by blocking the RNA–RNA base-pairing or protein–RNA binding ...
Mallory A. Havens, M. Hastings
semanticscholar   +1 more source

Meeting review : ESF workshop on "Impact of nucleic acid chemistry on gene function analysis: antisense, aptamers, ribozymes and RNAi" [PDF]

, 2008
The shortage of functional information compared to the abundance of sequence information characterizes today’s situation in functional genomics. For many years the knock-down of a gene’s product has been the most powerful way of analysing its function ...
Engels, Joachim W. (Prof. Dr.), Heermeier, Kathrin, Prydz, Hans, Reinhard-Rupp, Jutta   +3 more
core  

Cryptic Splicing of GAP43 mRNA is a Novel Hallmark of TDP‐43‐Associated ALS and AD

Advanced Science, EarlyView.
TDP‐43 dysfunction disrupts RNA processing, inducing cryptic exon 4a1 inclusion in GAP43 and reducing its protein levels. This aberrant splicing impairs axonal regeneration and contributes to neurodegeneration in ALS and AD. RNA‐seq of patient brains reveals GAP43 downregulation and 4a1 upregulation, identifying cryptic exon 4a1 as a potential ...
Mingming Yang, Qi Wang, Dongkun Kang, Shijia Wang, Yanli Jiang, Jian‐Zhi Wang, Chen Ming, Rong Liu, Jianlan Gu, Xiaochuan Wang   +9 more
wiley   +1 more source

Antisense Oligonucleotides Targeting Angiogenic Factors as Potential Cancer Therapeutics

Molecular Therapy: Nucleic Acids, 2019
Cancer is one of the leading causes of death worldwide, and conventional cancer therapies such as surgery, chemotherapy, and radiotherapy do not address the underlying molecular pathologies, leading to inadequate treatment and tumor recurrence ...
Bao T. Le, Prithi Raguraman, Tamer R. Kosbar, Susan Fletcher, Steve D. Wilton, Rakesh N. Veedu   +5 more
doaj  

Sarm1 haploinsufficiency or low expression levels after antisense oligonucleotides delay programmed axon degeneration

Cell Reports, 2021
Summary: Activation of the pro-degenerative protein SARM1 after diverse physical and disease-relevant injuries causes programmed axon degeneration. Original studies indicate that substantially decreased SARM1 levels are required for neuroprotection ...
Stacey Anne Gould, Jonathan Gilley, Karen Ling, Paymaan Jafar-Nejad, Frank Rigo, Michael Coleman   +5 more
doaj  

Dual targeting of MIR-208 and MIR-499 in the treatment of cardiac disorders [PDF]

, 2014
The present invention provides a method of treating or preventing cardiac disorders in a subject in need thereof by inhibiting the expression or function of both miR-499 and miR-208 in the heart cells of the subject. In particular, specific protocols for
Olson, Eric S., van Rooij, Eva
core   +1 more source

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

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

Biochemical prevention and treatment of viral infections – A new paradigm in medicine for infectious diseases [PDF]

, 2004
For two centuries, vaccination has been the dominating approach to develop prophylaxis against viral infections through immunological prevention. However, vaccines are not always possible to make, are ineffective for many viral infections, and also carry
Fang, Fang, Le Calvez, Hervé, Yu, Mang
core   +3 more sources

Delivery of Antisense Oligonucleotides to the Cornea

Nucleic Acid Therapeutics, 2020
Antisense oligonucleotides (ASOs) are synthetic nucleic acids that recognize complementary RNA sequences inside cells and modulate gene expression. In this study, we explore the feasibility of ASO delivery to the cornea. We used quantitative polymerase chain reaction to test the efficacy of a benchmark ASO targeting a noncoding nuclear RNA, Metastasis ...
Xin Gong, Venkateswara Mootha, David R. Corey, Frank Rigo, John D. Hulleman, Viet Q. Chau, Thahza P. Prakash, Jiaxin Hu, Rafael Ufret-Vincenty   +8 more
openaire   +4 more sources