Medicinal Research Reviews, EarlyView.ABSTRACT Photoremovable protecting groups (PPGs) offer a straightforward solution for the temporary inactivation of biologically active substrates and their subsequent controlled release by light irradiation. Their relatively easy design and mode of application have made them useful tools for studying dynamic biological processes in vitro and in vivo ...
Bence Kontra +3 more
wiley +1 more source
Serum Cardiac Troponin T Levels as a Therapy Response Marker in Tofersen‐Treated ALS
Muscle &Nerve, EarlyView.ABSTRACT Introduction/Aims Cardiac troponin T (cTnT) levels are elevated in the majority of persons with amyotrophic lateral sclerosis (ALS) and increase over time. Neurofilament light chain (NfL) is an established therapy response biomarker in ALS as superoxide dismutase1 (SOD1)‐ALS patients treated with the antisense oligonucleotide tofersen show a ...
Sarah Bernsen +7 more
wiley +1 more source
Correction of Aberrant Splicing of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene by Antisense Oligonucleotides [PDF]
, 1999 Kenneth J. Friedman +5 more
openalex +1 more source
Offline comprehensive liquid chromatography in combination with a Deoxyribonuclease I immobilized enzymatic reactor for selective screening of oligonucleotide mixtures [PDF]
, 2016 Alvarez Porebski, Piotr Wiktor +1 more
core +1 more source
mRNA fusion constructs serve in a general cell‐based assay to profile oligonucleotide activity [PDF]
, 2017 A cellular assay has been developed to allow measurement of the inhibitory activity of large numbers of oligonucleotides at the protein level. The assay is centred on an mRNA fusion transcript construct comprising of a full‐length reporter gene with a ...
Asselbergs, Fred +7 more
core
Progress in Aptamer Research and Future Applications
ChemistryOpen, EarlyView.This paper mainly describes the screening method of aptamer, the screening difficulties and improvement methods of small molecule aptamer, and the application and treatment of aptamer. It explores the latest progress and application of aptamers, and summarises them, thus indicating some problems that still exist in this field.
Song Liu +4 more
wiley +1 more source
Abrogation of the Fc gamma receptor IIA-mediated phagocytic signal by stem-loop Syk antisense oligonucleotides. [PDF]
, 1996 Masashi Matsuda +5 more
openalex +1 more source
Update on Gene Therapy in the Treatment of Hereditary Hearing Loss
Sensory Neuroscience, EarlyView.Gene therapy is a promising therapeutic approach for genetic disorders, including three major gene therapy strategies: gene replacement, gene suppression, and gene editing. After transmitting genes to the inner ear through appropriate pathways, repair the cochlea in order to achieve the goal of treating hereditary hearing loss.
Weiwei He +7 more
wiley +1 more source
Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.
, 1993 Z. Dominski, R. Kole
openalex +2 more sources