Results 51 to 60 of about 266,464 (233)
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 +6 more
core +1 more source
Repair of Aberrant Splicing in Growth Hormone Receptor by Antisense Oligonucleotides Targeting the Splice Sites of a Pseudoexon [PDF]
, 2010 Context: The GH receptor (GHR) pseudoexon 6 Psi defect is a frequent cause of GH insensitivity (GHI) resulting from a non-functioning GH receptor (GHR). It results in a broad range of phenotypes and may also be present in patients diagnosed as idiopathic
Clark, AJL +4 more
core +1 more source
Molecules, 2018 A novel 2′-F,4′-C-OMe–arabinouridine (araU) was successfully synthesized and introduced into oligonucleotides. The oligonucleotide containing 2′-F,4′-C-OMe–araU exhibited improved nuclease resistance and RNA ...
Xiao-Yang He +3 more
doaj +1 more source
Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review
European Journal of Cell Biology, 2023 Spinal muscular atrophy (SMA), the most common genetic cause of infantile death, is caused by a mutation in the survival of motor neuron 1 gene (SMN1), leading to the death of motor neurons and progressive muscle weakness.
Zorica Nakevska, Toshifumi Yokota
doaj
Molecular Therapy: Nucleic Acids, 2014 Antisense oligonucleotides complementary to RNA targets promise generality and ease of drug design. The first systemically administered antisense drug was recently approved for treatment and others are in clinical development. Chemical modifications that
Lykke Pedersen +3 more
doaj +1 more source
Polymerase-endonuclease amplification reaction for large-scale enzymatic production of antisense oligonucleotide [PDF]
, 2009 Synthetic oligonucleotides are contaminated with highly homologous failure sequences. Oligonucleotide synthesis is difficult to scale up because it requires expensive equipments, hazardous chemicals, and tedious purification process.
Deming Gou, Xiaolong Wang
core +1 more source
Antisense Oligonucleotide Therapies for Neurodegenerative Diseases.
Annual Review of Neuroscience, 2019 Antisense oligonucleotides represent a novel therapeutic platform for the discovery of medicines that have the potential to treat most neurodegenerative diseases.
C. Bennett, A. Krainer, D. Cleveland
semanticscholar +1 more source
Voltage-gated calcium channel and antisense oligonucleotides thereto [PDF]
, 1998 An antisense oligonucleotide of 10 to 35 nucleotides in length that can hybridize with a region of the .alpha..sub.1 subunit of the SA-Cat channel gene DNA or mRNA is provided, together with pharmaceutical compositions containing and methods utilizing ...
Barry, Elizabeth L. R. +3 more
core +1 more source
Nucleic Acid Carriers Based on Precise Polymer Conjugates [PDF]
, 2011 Polymer polydispersity, random conjugation of functional groups, and poorly understood structure–activity relationships have constantly hampered progress in the development of nucleic acid carriers.
Troiber, Christina, Wagner, Ernst
core +1 more source
Frontiers in Pharmacology, 2019 The worldwide spread of multidrug-resistant Mycobacterium tuberculosis strains prompted the development of new strategies to combat tuberculosis, one of which is antisense therapy based on targeting bacterial mRNA by oligonucleotide derivatives. However,
Yulia V. Skvortsova +7 more
doaj +1 more source