Results 101 to 110 of about 47,774 (288)

Therapy for Myhre Syndrome: Goals, Misconceptions, and Current Agents

American Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.
ABSTRACT Myhre Syndrome (MYHRS, MIM #139210) is a rare, multisystem connective tissue disorder caused by recurrent heterozygous gain‐of‐function pathogenic variants in the SMAD4 gene, a key player in TGF‐β signaling and a regulator of extracellular matrix homeostasis.
Alessandro De Falco, Alfonso Manuel D'Alessio, Nicola Brunetti‐Pierri   +2 more
wiley   +1 more source

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
doaj   +1 more source

Antisense strategies: delivery of oligonucleoides by nonviral nanoparticles

, 2015
International audienceGene silencing approaches have demonstrated high promise for the development of new treatments of diseases caused by the abnormal overexpression of a known gene.
Vauthier, Christine, Malvy, Claude, Claude Malvy, Christine Vauthier   +3 more
core   +1 more source

Therapeutic Applications of Stimuli‐Based Release and Engineering of Extracellular Vesicles

Advanced NanoBiomed Research, EarlyView.
This review summarizes the effects of endogenous and exogenous stimuli, their effects on the natural release of extracellular vesicles, as well as their uptake and release. It also gives an overview of stimuli‐responsive EVs and their therapeutic applications. Extracellular vesicles (EVs), nano‐ to microsized lipid bilayer membrane‐bound particles, are
Gloria Kemunto, Kristen Dellinger
wiley   +1 more source

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
doaj   +1 more source

Exosome-Mediated Mitochondrial Delivery of Antisense Oligonucleotides

Nucleic Acid Therapeutics
We present a general method for in-cellulo delivery of 2′- O -methyl (2′-OMe) RNA oligonucleotides (oligos) to mitochondria for antisense applications, with potential for implementation in other mitochondrial DNA (mtDNA)-targeted therapies.
Dora von Trentini, Ivan J. Dmochowski
openaire   +3 more sources

Auf dem Weg zu chromoselektiven Transformationen in biologischen Systemen: Perspektiven und Herausforderungen

Angewandte Chemie, EarlyView.
Die Kontrolle biologischer Systeme mit organischen Chromophoren hat sich zu einer vielversprechenden Strategie entwickelt und findet Anwendung von der chemischen Biologie bis hin zur Medizin. Die Komplexität von In‐vivo‐Systemen lässt sich jedoch nicht mit nur einer einzigen Lichtwellenlänge adäquat abbilden.
Nadja A. Simeth
wiley   +1 more source

Novel cationic amphiphiles as delivery agents for antisense oligonucleotides

, 1999
There has been great interest recently in therapeutic use of nucleic acids including genes, ribozymes and antisense oligonucleotides. Despite recent improvements in delivering antisense oligonucleotides to cells in culture, nucleic acid-based therapy is ...
Fisher, M., Kang, S. H., Alahari, S. K., Regan, S. L., Kole, R., Janout, V., Hoon, Yoo, Short, S. M., Juliano, R. L., DeLong, R. K.   +9 more
core   +1 more source

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

Toward Chromoselective Transformations in Biological Systems: Perspectives and Challenges

Angewandte Chemie International Edition, EarlyView.
Controlling biological systems with small‐molecule chromophores has evolved into a powerful strategy and is applied from chemical biology to medicine. However, the complexity of in vivo systems cannot be matched by a single wavelength of light. Developing methods to combine and individually control multiple chromophores is crucial.
Nadja A. Simeth
wiley   +1 more source