Splicing therapeutics in SMN2 and APOB [PDF]
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 ...
Khoo, B., Krainer, A. R.
core
Modulation of swimming in the gastropod Melibe leonina by nitric oxide [PDF]
Nitric oxide (NO) is a gaseous intercellular messenger produced by the enzyme nitric oxide synthase. It has been implicated as a neuromodulator in several groups of animals, including gastropods, crustaceans and mammals.
Newcomb, James M. +1 more
core +1 more source
Immaturity of the neuromuscular junction in spinal muscular atrophy mouse models. [PDF]
Tabares L +4 more
europepmc +1 more source
RNA-binding proteins TDP-43 and FUS promote R-loop resolution and regulate transcription termination. [PDF]
Zhao DY +8 more
europepmc +1 more source
RNA biomarkers in spinal muscular atrophy: enhancing pathogenesis understanding and guiding precision medicine. [PDF]
Alberti C +4 more
europepmc +1 more source
Fibro-adipogenic progenitor cells from murine SMA muscles are intrinsically adipogenic. [PDF]
Luo YE +5 more
europepmc +1 more source
Spliceosomal Sm core assembly: AlphaFold 3 predicted structure and phosphorylation-dependent regulation of the human 6S complex. [PDF]
Grimmler M +3 more
europepmc +1 more source
Novel Translational Concept: Axon-to-Muscle Exosomal Signaling as an Emerging Therapeutic Target in Spinal Muscular Atrophy. [PDF]
Fajkić A +8 more
europepmc +1 more source
Fast-Dissolving Sodium Alginate-Based Microneedle Patch Integrating Tranilast and Glabridin-Loaded Nanoparticles for Dual-Action Hypertrophic Scar Therapy. [PDF]
Younas A +9 more
europepmc +1 more source
SUMO2 promotes histone pre-mRNA processing by stabilizing histone locus body interactions and facilitating U7 snRNP assembly. [PDF]
He S +10 more
europepmc +1 more source

