Results 201 to 210 of about 1,038,708 (317)
ABSTRACT Objective Facioscapulohumeral muscular dystrophy (FSHD) is one of the most debilitating and common muscular dystrophies. Despite its severity, no approved therapy exists for FSHD patients. However, several therapeutic candidates are currently under development, and some have recently entered clinical trials, marking the need for reliable ...
Mustafa Bilal Bayazit +11 more
wiley +1 more source
Systematic identification of variant-specific RNA structure-small molecule interactions exemplified by RNA G-quadruplexes. [PDF]
Miyashita E +12 more
europepmc +1 more source
ABSTRACT Objective Neurochemical levels measured by brain MR spectroscopy (MRS) have been proposed as endpoints for clinical trials in early‐stage spinocerebellar ataxia (SCA) trials. We tested their trial‐readiness by quantifying neurochemicals in three affected brain regions in early‐stage cohorts of SCA2 and SCA3, examining their reproducibility in ...
James M. Joers +19 more
wiley +1 more source
RNA structure modulates Cas13 activity and enables mismatch detection. [PDF]
Larsen BB +16 more
europepmc +1 more source
A Hitchhiker's guide to RNA-RNA structure and interaction prediction tools. [PDF]
Tieng FYF +5 more
europepmc +1 more source
Cognitive and Neuroimaging Divergence Between Juvenile and Adult FUS Amyotrophic Lateral Sclerosis
ABSTRACT Objective Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by progressive motor neuron degeneration. Fused in sarcoma (FUS)‐associated juvenile ALS (jALS) represents a distinct and aggressive subgroup with rapid deterioration and poor prognosis.
Alexandra V. Jürs +7 more
wiley +1 more source
TiRNA: a coarse-grained method with temperature and ion effects for RNA structure folding and prediction. [PDF]
Wang X +5 more
europepmc +1 more source
Objective JAK inhibitors (JAKis) have shown promising effects in early‐phase studies of SSc. We aimed to assess the safety and explore effectiveness of JAKis compared to conventional immunosuppressants in SSc. Methods A longitudinal retrospective study of the EUSTAR cohort was performed.
Stefano Di Donato +27 more
wiley +1 more source
DeepRMSF: a deep learning-based automated approach for predicting atomic-level flexibility in RNA structure. [PDF]
Feng C +5 more
europepmc +1 more source
A 3D anisotropic hydrogel derived from heart extracellular matrix guides cytoskeletal alignment and nuclear remodeling in reprogrammed cardiomyocyte‐like cells. This study reveals how matrix alignment modulates nuclear envelope dynamics and chromatin state, triggering transcriptional and functional maturation.
Seung Ju Seo +7 more
wiley +1 more source

