Separation of PS diastereomers in GalNAc-conjugated siRNA-a comparative study of chromatographic and ion mobility mass spectrometry approaches. [PDF]
Bill MK +8 more
europepmc +1 more source
New cell lines expanding the diversity of Ewing sarcoma models
What's new? A chromosomal translocation between the EWSR1 and ETS genes is the key oncogenic driver in Ewing sarcoma. EWSR1::FLI1, the most frequent fusion, is subdivided into different fusion types with unclear prognostic relevance. Here, the authors describe the establishment and characterization of four new, intrinsically immortal, patient‐derived ...
Maximilian Kerkhoff +13 more
wiley +1 more source
MiRNA-based drugs: challenges and delivery strategies. [PDF]
Wang Z +5 more
europepmc +1 more source
ABSTRACT Background Doxorubicin (DOX) is a highly effective chemotherapeutic agent whose use can cause respiratory toxicity, increasing patient fatigue and negatively impacting quality of life and survival. These adverse effects occur due to diaphragm muscle mitochondrial accumulation of DOX, where it causes reactive oxygen species production and iron ...
Ashley J. Smuder +4 more
wiley +1 more source
Advances in RNA-based therapeutics: current breakthroughs, clinical translation, and future perspectives. [PDF]
Makkar SK.
europepmc +1 more source
Current Trends in Duchenne Muscular Dystrophy Research and Therapy: 3D Cardiac Modelling
ABSTRACT Duchenne muscular dystrophy (DMD), caused by dystrophin deficiency, presents a multifaceted challenge that affects both skeletal muscle function and cardiomyocyte homeostasis, causing progressive degeneration and life‐threatening cardiac complications by adolescence.
Marta Przymuszała +3 more
wiley +1 more source
Synthesis, Biological Activity, and Molecular Dynamics Simulations of LNA-Charge Neutral Linkages for Enhanced Splice-Switching Antisense Oligonucleotides. [PDF]
Kennett A +11 more
europepmc +1 more source
ABSTRACT The interaction between mutated leucine‐rich repeat kinase 2 (LRRK2) and the death adaptor protein FADD accounts for apoptotic death of dopaminergic neurons in familial Parkinson's disease (PD) driven by LRRK2 mutations. Disrupting this pathogenic interaction using constrained peptides is a promising therapeutic strategy to mitigate apoptotic ...
Yaochao Zheng +6 more
wiley +1 more source
A rare 5'UTR variant in SEC24D reveals translational dysfunction in osteogenesis imperfecta: a roadmap for RNA therapeutic rescue. [PDF]
Essawi O +7 more
europepmc +1 more source

