When <i>HERG</i>-caused LQT2 encounters antisense oligonucleotide: is exon 6 skipping therapy plausible? [PDF]
Zheng Z, Song Y.
europepmc +1 more source
Through our exploration of the synergistic effects of drug combinations for potential clinical applications, we found that steroids potentiate the anticancer effects of etoposide, a topoisomerase II inhibitor. Steroids, when combined with etoposide, increase DNA double‐strand breaks not via DNA repair inhibition but transcriptional modulation ...
Ying Zhao+11 more
wiley +1 more source
Systematic analysis of genetic and phenotypic characteristics reveals antisense oligonucleotide therapy potential for one-third of neurodevelopmental disorders. [PDF]
Wijnant KN, Nadif Kasri N, Vissers LELM.
europepmc +1 more source
Longitudinal Visual Biomarkers in Dominant Optic Atrophy: A Systematic Review and Meta‐Analysis
ABSTRACT Background Dominant Optic Atrophy (DOA) causes slowly progressive visual decline usually beginning in childhood. As new therapies come to clinical trial, the choice of biomarkers to be used as clinical trial endpoints has become a critical question to be addressed. Methods We undertook a systematic review and meta‐analysis of studies reporting
Christopher A. Ovens+2 more
wiley +1 more source
Robust Photocleavable Linkers for DNA Synthesis: Enabling Visible Light-Triggered Antisense Oligonucleotide Release in 3D DNA Nanocages. [PDF]
Leung HM+7 more
europepmc +1 more source
Briefly, SLC30A4‐AS1 is upregulated in senescent PDLSCs mediated by inflammation. SLC30A4‐AS1 binds the splicing factor SRSF3 to affect the alternative splicing of TP53BP1 and alter the expression of its transcript TP53BP1‐204. Knockdown of TP53BP1‐204 expression can improve the senescence of PDLSCs and improve the osteogenic differentiation ability of
Mei Xu+11 more
wiley +1 more source
Evaluation of an Antisense Oligonucleotide Targeting CAG Repeats: A Patient-Customized Therapy Study for Huntington's Disease. [PDF]
Ocampo-Ortega SA+10 more
europepmc +1 more source
Abstract Lipoprotein(a) [Lp(a)] is a genetically determined, causal risk factor for atherosclerotic cardiovascular disease (ASCVD) and calcific aortic valve disease (CAVD). Despite robust evidence from epidemiological and genetic studies, Lp(a) remains underrecognised in clinical practice due to challenges in measurement, lack of guideline familiarity ...
Alexander C. Razavi+4 more
wiley +1 more source
Patients with transthyretin amyloid cardiomyopathy (ATTR‐CM) were excluded from randomized clinical trials (RCTs) evaluating sodium‐glucose cotransporter 2 inhibitors (SGLT2i); as a result, the effects of these agents on this population remain underexplored.
Paschalis Karakasis+7 more
wiley +1 more source
Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells [PDF]
Suzuki N.
europepmc +2 more sources