Results 61 to 70 of about 6,268,347 (286)

Lysosomes, Lysosomal Storage Diseases, and Inflammation [PDF]

open access: yesJournal of Inborn Errors of Metabolism and Screening, 2016
Lysosomes were originally described in the early 1950s by de Duve who was also the first to recognize the importance of these organelles in human disease. We know now that lysosomes are involved in numerous biological processes, and abnormalities in lysosomal function may result in a broad range of diseases. This review will briefly discuss the role of
openaire   +4 more sources

Fabry Disease (A Kind of Lysosomal Storage Disease) [PDF]

open access: yes, 2012
How to Cite this Article: Karimzadeh P. Fabery Disease (A kind of Lysosomal Storage Disease). Iran J Child Neurol Autumn 2012; 6:4(suppl. 1):6.
KARIMZADEH, Parvaneh
core   +1 more source

Fusogenic RNA Nanomodules for Fusion‐Mediated and Multiplexed siRNA Delivery

open access: yesAdvanced Functional Materials, EarlyView.
A fusogenic lipid‐layered RNA nanomodules (L‐CRAMs) enable high‐capacity and long‐lasting siRNA delivery through membrane fusion. These nanomodules carry exceptionally large siRNA payloads, avoid conventional endosomal uptake, and release multiple functional siRNAs through Dicer‐mediated processing.
Sunghyun Moon   +5 more
wiley   +1 more source

Recent advances in gene therapy for lysosomal storage disorders [PDF]

open access: yes, 2015
David PW Rastall,1 Andrea Amalfitano1,2 1Department of Microbiology and Molecular Genetics, 2Department of Pediatrics, College of Osteopathic Medicine, Michigan State University, East Lansing, MI, USA Abstract: Lysosomal storage disorders (LSDs) are a ...
Amalfitano A, Rastall DP
core  

A Holistic Platform Approach for Developing Robust Pulmonary mRNA Delivery: Integrating Novel Phenolic Acid‐Derived Ionizable Lipids and Device Engineering

open access: yesAdvanced Functional Materials, EarlyView.
An integrated pulmonary mRNA delivery platform combining novel biodegradable syringic acid‐derived ionizable lipids, design‐of‐experiments formulation optimization, and vibrating‐mesh nebulizer engineering enabled stable aerosolization and efficient lung delivery.
Neha Kaushal   +21 more
wiley   +1 more source

A terpene nucleoside from M. tuberculosis induces lysosomal lipid storage in foamy macrophages

open access: yesThe Journal of Clinical Investigation, 2023
Induction of lipid-laden foamy macrophages is a cellular hallmark of tuberculosis (TB) disease, which involves the transformation of infected phagolysosomes from a site of killing into a nutrient-rich replicative niche.
Melissa Bedard   +22 more
doaj   +1 more source

Microfluidic Nano‐Assembly of Red‐Blood‐Cell (RBC) Lipids and Components for Engineering Extracellular Vesicles

open access: yesAdvanced Healthcare Materials, EarlyView.
Engineered red blood cell‐derived extracellular vesicles (eRBCEVs) are synthesized via controlled microfluidic assembly from native RBC lipids, enabling tunable encapsulation of proteins, nucleic acids, nanoparticles, and viral vectors. The platform demonstrates reproducible nanoscale architecture, preserved membrane composition, and functional cargo ...
Chiranth K. Nagaraj   +23 more
wiley   +1 more source

Neurodegeneration Upon Dysfunction of Endosomal/Lysosomal CLC Chloride Transporters

open access: yesFrontiers in Cell and Developmental Biology, 2021
The regulation of luminal ion concentrations is critical for the function of, and transport between intracellular organelles. The importance of the acidic pH in the compartments of the endosomal-lysosomal pathway has been well-known for decades.
Shroddha Bose   +4 more
doaj   +1 more source

From Cell‐Derived Vesicles to Hybrid Nanovectors: Biological Membranes as Functional Blueprints for Gene Delivery

open access: yesAdvanced Healthcare Materials, EarlyView.
Biologically derived and hybrid nonviral nanovectors are examined as distinct but convergent design approaches. Integrating synthetic components with biologically functionalized membranes allows efficient interactions with complex cellular environments.
Clara Baldari   +10 more
wiley   +1 more source

Development of a fluorometric microtiter plate based enzyme assay for MPS IVA (Morquio type A) using dried blood spots

open access: yesMolecular Genetics and Metabolism Reports, 2014
Mucopolysaccharidosis type IVA or Morquio type-A disease is a hereditary lysosomal storage disorder caused by deficient activity of the lysosomal enzyme N-acetylgalactosamine-6-sulfate sulfatase (GALNS). The disease is caused by lysosomal accumulation of
Anirudh J. Ullal   +2 more
doaj   +1 more source

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