Results 221 to 230 of about 123,112 (260)

Expanding the Interface: Overlooked Dermatologic Disorders With Ocular Involvement

JEADV Clinical Practice, EarlyView.
A George
wiley   +1 more source

Enhanced delivery of α-glucosidase for Pompe disease by ICAM-1-targeted nanocarriers: comparative performance of a strategy for three distinct lysosomal storage disorders

, 2011
Janet Hsu, Laura Northrup, Tridib Kumar Bhowmick, Silvia Muro   +3 more
openalex   +2 more sources

Migrasomes, Matrix‐Bound Nanovesicles, and More: Messengers in the Matrix

PROTEOMICS, EarlyView.
ABSTRACT Extracellular vesicles (EVs) and particles (EPs) are diverse micro‐ and nanoparticles that circulate in bodily fluids and can attach to, or be deposited onto, the extracellular matrix (ECM) and other surfaces. To date, the nomenclature and classification of matrix‐bound or matrix‐associated EVs and EPs (MEVPs) have been unclear, largely due to
Anna V. Kolesov, Natalie Reitz, Marley Dewey, Colin L. Hisey   +3 more
wiley   +1 more source

An improved purification method for the lysosomal storage disease protein β-glucuronidase produced in CHO cells. [PDF]

Protein Expr Purif, 2017
Fratz-Berilla EJ, Ketcham SA, Parhiz H, Ashraf M, Madhavarao CN.   +4 more
europepmc   +1 more source

Newborn Screening of Lysosomal Storage Diseases, Including Mucopolysaccharidoses

, 2017
Sujin Kim
openalex   +1 more source

Lysosomal storage diseases as a complex pathophysiological and clinical problem - part one

Karolina Mikołajczak
openalex   +2 more sources

Proteomics Insights Into Lysosome Biogenesis and Maturation

PROTEOMICS, EarlyView.
ABSTRACT Lysosomes constitute the main degradative organelle of most eukaryotic cells and are capable of breaking down a wide spectrum of biomolecules, including proteins, lipids, glycans, and DNA/RNA. They play crucial roles in the regulation of cellular homeostasis, acting as metabolic signaling centers for the correlation of nutrient availability ...
Katharina Hirn, Sofía Fajardo‐Callejón, Dominic Winter   +2 more
wiley   +1 more source

Transforming Pediatric Rare Disease Drug Development: Enhancing Clinical Trials and Regulatory Evidence With Virtual Patients

CPT: Pharmacometrics &Systems Pharmacology, EarlyView.
ABSTRACT Drug development in pediatric rare diseases is complicated by practical and ethical constraints on clinical trial design, stemming from small, highly heterogeneous, and vulnerable patient populations. Virtual patients (VPs) created with machine‐learning (ML), mechanistically driven computational approaches, or hybrids thereof, have the ...
Fianne Sips, Marco Virgolin, Giuseppe Pasculli, Federico Reali, Alessio Paris, Annette Janus, Yann Godfrin, Daniel Röshammar, Luca Marchetti, Jane Knöchel   +9 more
wiley   +1 more source

Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing

Small, EarlyView.
The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa, Mamata Karmacharya, Hyunmin Choi, Sumit Kumar, Yoon‐Kyoung Cho   +4 more
wiley   +1 more source

Performance of the Four-Plex Tandem Mass Spectrometry Lysosomal Storage Disease Newborn Screening Test: The Necessity of Adding a 2nd Tier Test for Pompe Disease. [PDF]

Int J Neonatal Screen, 2018
Chiang SC, Chen PW, Hwu WL, Lee AJ, Chen LC, Lee NC, Chiou LY, Chien YH.   +7 more
europepmc   +1 more source