Results 91 to 100 of about 270,275 (243)
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 +3 more
doaj +1 more source
Splicing therapeutics in SMN2 and APOB
, 2009 Splicing therapeutics are defined as the deliberate modification of RNA splicing to achieve therapeutic goals. Various techniques for splicing therapeutics have been described, and most of these involve the use of antisense oligonucleotide-based ...
Krainer, AR +3 more
core
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
Molecular Therapy: Nucleic Acids, 2019 Eluforsen (previously known as QR-010) is a 33-mer 2′-O-methyl modified phosphorothioate antisense oligonucleotide targeting the F508del mutation in the gene encoding CFTR protein of cystic fibrosis patients.
Jaeah Kim +6 more
doaj +1 more source
Antisense oligonucleotides or oligonucleotide analogues [PDF]
, 2013 The present invention relates to antisense oligonucleotides or oligonucleotide analogues adapted to target pre-mRNA which encodes GNB3s and prevent aberrant splicing events.
Lester, Douglas H.
core
Oligonucleotide-peptide conjugates as potential antisense agents
, 1999 Oligonucleotide-peptide conjugates have several applications, including their potential use as improved antisense agents for interfering with the RNA function within cells.
Zoe A Shabarova +13 more
core +1 more source
ANTISENSE MEDIATED DYSTROPHIN READING FRAME RESTORATION
, 2010 Exon skipping using antisense oligonucleotides (AONs) has successfully been used to reframe the mRNA in various DMD (Duchenne muscular dystrophy) patients carrying deletions and in the mdx mouse model.
Spitali, Pietro
core
Molecular Therapy: Nucleic Acids, 2017 Antisense oligonucleotide (AON) therapeutics offer new avenues to pursue clinically relevant targets inaccessible with other technologies. Advances in improving AON affinity and stability by incorporation of high affinity nucleotides, such as locked ...
Annie Moisan +17 more
doaj +1 more source
Alimentary Pharmacology and Therapeutics, 2016 In a phase 2 study, mongersen, an oral antisense oligonucleotide targeting Smad7, was effective in inducing clinical remission in approximately 60% of patients with active Crohn's disease (CD).
G. Monteleone +7 more
semanticscholar +1 more source
Analytical HPLC analysis of purified antisense oligonucleotide.
, 2013 Sample preparation: 25 µL purified antisense oligonucleotide; Column: DNAPac PA-100 (4/250); Flow rate: 1 ml/min; Buffer A: 10 mM NaClO4+1 mM Tris; Buffer B: 300 mM NaClO4+1 mM Tris; Gradient: 10–70% B, 7.6 CV.
Shuang-yong Xu (14559) +2 more
core +1 more source