Results 101 to 110 of about 83,454 (234)

Electroporation Enhanced Effect of Dystrophin Splice Switching PNA Oligomers in Normal and Dystrophic Muscle

Molecular Therapy: Nucleic Acids, 2015
Peptide nucleic acid (PNA) is a synthetic DNA mimic that has shown potential for discovery of novel splice switching antisense drugs. However, in vivo cellular delivery has been a limiting factor for development, and only few successful studies have been
Camilla Brolin, Takehiko Shiraishi, Pernille Hojman, Thomas O Krag, Peter E Nielsen, Julie Gehl   +5 more
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Locked nucleic acid: tighter is different [PDF]

, 2013
This viewpoint briefly reviews the impact of Locked Nucleic Acid (LNA) oligonucleotides, first described in a ChemComm paper in 1998. A number of unique applications in oligonucleotide biotechnology have been made possible by the high binding affinity ...
Watts, Jonathan
core   +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
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Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo

Nature Communications
The most common genetic cause of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) is an intronic G4C2 repeat expansion in C9orf72.
Liam Kempthorne, Deniz Vaizoglu, Alexander J. Cammack, Mireia Carcolé, Martha J. Roberts, Alla Mikheenko, Alessia Fisher, Pacharaporn Suklai, Bhavana Muralidharan, François Kroll, Thomas G. Moens, Lidia Yshii, Stijn Verschoren, Benedikt V. Hölbling, Francisco C. Moreira, Eszter Katona, Rachel Coneys, Paula de Oliveira, Yong-Jie Zhang, Karen Jansen, Lillian M. Daughrity, Alexander McGown, Tennore M. Ramesh, Ludo Van Den Bosch, Gabriele Lignani, Ahad A. Rahim, Alyssa N. Coyne, Leonard Petrucelli, Jason Rihel, Adrian M. Isaacs   +29 more
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Antisense and Functional Nucleic Acids in Rational Drug Development

Antibiotics
This review is focused on antisense and functional nucleic acid used for completely rational drug design and drug target assessment, aiming to reduce the time and money spent and increase the successful rate of drug development. Nucleic acids have unique
Robert Penchovsky, Antoniya V. Georgieva, Vanya Dyakova, Martina Traykovska, Nikolet Pavlova   +4 more
doaj   +1 more source

Micro-RNAS that control myosin expression and myofiber identity [PDF]

, 2015
The present invention relates to the identification of two microRNAs, miR-499 and miR-208b, that repress fast skeletal muscle contractile protein genes. Expression of miR-499 and/or miR-208b can be used to repress fast fiber genes and activate slow fiber
Olson, Eric N., van Rooij, Eva
core   +1 more source

Inhibition of the tyrosine phosphatase SHP-2 suppresses angiogenesis in vitro and in vivo [PDF]

, 2007
Endothelial cell survival is indispensable to maintain endothelial integrity and initiate new vessel formation. We investigated the role of SHP-2 in endothelial cell survival and angiogenesis in vitro as well as in vivo.
Alavi A, Barbara Walzog, Bennett AM, Brooks PC, Chan RJ, Chang F, Chen J, Christian Plank, Crompton T, Cunnick JM, Distler JH, Florian Krötz, Frisch SM, Hadari YR, Hae-Young Sohn, Hanna Mannell, Hu Y, Huang P, Ivins Zito C, Kontaridis MI, Krotz F, Krotz F, Krotz F, Krotz F, Leopold Groesser, Munoz-Chapuli R, Neel BG, Neel BG, Nicole Hellwig, Noguchi T, Nor JE, Ong SH, O’Brien CD, Papetti M, Plank C, Qi JH, Qi JH, Qu CK, Ren Y, Saxton TM, Shi ZQ, Slater EP, Tang TL, Thakker GD, Tomanek RJ, Torsten Gloe, Ulrich Pohl, Wu CJ, Yart A, Zhang SQ, Zhang SQ   +50 more
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The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders

Biomedicines, 2017
Antisense Oligonucleotides (ASOs) represent very attractive therapeutic compounds for the treatment of numerous diseases. The antisense field has remarkably progressed over the last few years with the approval of the first antisense drugs and with ...
Philippine Aupy, Lucía Echevarría, Karima Relizani, Aurélie Goyenvalle   +3 more
doaj   +1 more source

Genetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy [PDF]

, 2016
Accurate definition of genetic mutations causing Duchenne muscular dystrophy (DMD) has always been relevant in order to provide genetic counseling to patients and families, and helps to establish the prognosis in the case where the distinction between ...
Bello, Luca, Pegoraro, Elena
core  

Bolaamphiphile-based nanocomplex delivery of phosphorothioate gapmer antisense oligonucleotides as a treatment for Clostridium difficile

International Journal of Nanomedicine, 2016
John P Hegarty,1 Jacek Krzeminski,2 Arun K Sharma,2 Diana Guzman-Villanueva,3 Volkmar Weissig,3 David B Stewart Sr1 1Deparment of Surgery, Pennsylvania State University College of Medicine Hershey, PA, USA; 2Department of Pharmacology, Penn State ...
Hegarty JP, Krzeminski J, Sharma AK, Guzman-Villanueva D, Weissig V, Stewart DB Sr   +5 more
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