Non‐animal extracellular vesicles as bioactive‐compound carriers: compositional characteristics, engineering strategies, and biofunctions. Abstract Animal‐derived extracellular vesicles (EVs) were widely used for bioactive compound delivery but constrained by complex origins, potential immunogenicity, and pathogen contamination.
Jiahao Yu +6 more
wiley +1 more source
Characterization of Dried Blood Spot Quality Control Materials for Lysosomal Enzyme Activity Assays Using Digital Microfluidic Fluorometry to Detect Lysosomal Storage Disorders in Newborns. [PDF]
Dantonio P +9 more
europepmc +1 more source
The Human Biomarker Navigator integrates the disease continuum, biomarker dynamics, cross‐organ biomarker networks, biomarker classification, and technology‐driven paradigms. It maps how biomarkers link multi‐system physiology and pathology across the nervous, respiratory, endocrine, circulatory, immune, digestive, urinary, reproductive, and ...
Meng‐Yao Li +29 more
wiley +1 more source
Assessment of the reliability, responsiveness, and meaningfulness of the scale for the assessment and rating of ataxia (SARA) for lysosomal storage disorders. [PDF]
Park J +22 more
europepmc +1 more source
ATP Citrate Lyase in Metabolic Disease: Mechanistic Insights and Clinical Potential
ATP citrate lyase (ACLY) is a central metabolic hub that diverts mitochondrial citrate to fuel de novo lipogenesis, cholesterol biosynthesis, and protein acetylation. Given its robust correlation with pathological changes in multiple human diseases, ACLY inhibitors featuring distinct pharmacological strengths have been developed for therapeutic ...
Wenbiao Wang +5 more
wiley +1 more source
Novel pathomechanistic insights into lysosomal storage disorders: how neuron-intrinsic cGAS-STING signaling drives disease progression. [PDF]
Frosch M, Prinz M.
europepmc +1 more source
Heart failure in two male patients with late‐onset Fabry mutation (IVS4 + 919G > A)
ESC Heart Failure, Volume 12, Issue 2, Page 1508-1513, April 2025.
Xufei Yang +3 more
wiley +1 more source
GSK3β drives early diabetic tubulopathy via TFEB‐mediated mitochondrial dysfunction
Mitochondrial dysfunction, including mitochondrial biogenesis, mitophagy, dynamics and oxidative stress, occurs in the early stages of diabetic tubulopathy, which precede proteinuria and renal histological changes. GSK3β is a potential novel biomarker for the prediction of diabetic tubulopathy.
Lan Yao +10 more
wiley +1 more source
Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey. [PDF]
Corazolla EM +8 more
europepmc +1 more source

