Results 61 to 70 of about 154,911 (267)

β‐Catenin/c‐Myc Axis Modulates Autophagy Response to Different Ammonia Concentrations

open access: yesAdvanced Biology, Volume 9, Issue 3, March 2025.
Ammonia, detoxified by the liver into urea and glutamine, impacts autophagy differently at varying levels. Low ammonia activates autophagy via c‐Myc and β‐catenin, while high levels suppress it. Using Huh7 cells and Spf‐ash mice, c‐Myc's role in cytoprotective autophagy is revealed, offering insights into hyperammonemia and potential therapeutic ...
S. Sergio   +11 more
wiley   +1 more source

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

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

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

Gene therapy in lysosomal diseases

open access: yesBiomedicine & Pharmacotherapy, 2000
Lysosomal storage diseases are monogenic metabolic disorders resulting from a deficiency in intralysosomal enzymes involved in macromolecule catabolism. Various groups have been delineated according to the affected pathway and the accumulated substrate: mucopolysaccharidoses, lipidoses, glycoproteinoses and glycogenosis type II. Their clinical severity
C, Caillaud, L, Poenaru
openaire   +2 more sources

Lysosomal targeting of liposomes with acidic pH and Cathepsin B induces protein aggregate clearance

open access: yesCell Communication and Signaling
The autophagy-lysosomal pathway is a cellular degradation mechanism that regulates protein quality by eliminating aggregates and maintaining normal protein function.
Minsol Jeon   +6 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

Rare lysosomal disease registries: lessons learned over three decades of real-world evidence. [PDF]

open access: yesOrphanet J Rare Dis, 2022
Mistry PK   +6 more
europepmc   +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

TRPML1 activation ameliorates lysosomal phenotypes in CLN3 deficient retinal pigment epithelial cells

open access: yesScientific Reports
Mutations in the lysosomal membrane protein CLN3 cause Juvenile Neuronal Ceroid Lipofuscinosis (JNCL). Activation of the lysosomal ion channel TRPML1 has previously been shown to be beneficial in several neurodegenerative disease models.
D. Wünkhaus   +13 more
doaj   +1 more source

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