Results 101 to 110 of about 238,474 (313)

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

Orphanet J Rare Dis, 2022
Mistry PK, Kishnani P, Wanner C, Dong D, Bender J, Batista JL, Foster J.   +6 more
europepmc   +1 more source

Shellac‐Mediated Assembly of Nanoparticles for mRNA Delivery

Advanced Healthcare Materials, EarlyView.
A shellac‐mediated nanoparticle assembly strategy is presented, involving the complexation of mRNA and low‐molecular‐weight cationic molecules (e.g., polyethyleneimine, chitosan, lipids, and poly‐l‐lysine), followed by capping with shellac. This nanoparticle platform enables mRNA transfection following intravenous injection and facilitates gene editing
Meizhang Lu, Jingqu Chen, Zhixing Lin, Wanjun Xu, Tianzheng Wang, Shiyao Li, Die Yang, Frank Caruso   +7 more
wiley   +1 more source

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

Scientific 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, R. Tang, K. Nyame, N. N. Laqtom, M. Schweizer, A. Scotto Rosato, E. K. Krogsæter, C. Wollnik, M. Abu-Remaileh, C. Grimm, G. Hermey, R. Kuhn, D. Gruber-Schoffnegger, S. Markmann   +13 more
doaj   +1 more source

Fluorogenic Substrates for Visualizing Acidic Organelle Enzyme Activities.

PLoS ONE, 2016
Lysosomes are acidic cytoplasmic organelles that are present in all nucleated mammalian cells and are involved in a variety of cellular processes including repair of the plasma membrane, defense against pathogens, cholesterol homeostasis, bone remodeling,
Fiona Karen Harlan, Jason Scott Lusk, Breanna Michelle Mohr, Anthony Peter Guzikowski, Robert Hardy Batchelor, Ying Jiang, John Joseph Naleway   +6 more
doaj   +1 more source

Reprogramming of lysosomal gene expression by interleukin-4 and Stat6. [PDF]

, 2013
BACKGROUND: Lysosomes play important roles in multiple aspects of physiology, but the problem of how the transcription of lysosomal genes is coordinated remains incompletely understood.
Bartlett, NW, Brignull, LM, Czimmerer, Z, Daniel, B, Johnston, SL, Meira, LB, Nagy, L, Nohturfft, A, Saidi, H, Villela, I   +9 more
core   +2 more sources

Bacteria‐Responsive Nanostructured Drug Delivery Systems for Targeted Antimicrobial Therapy

Advanced Materials, EarlyView.
Bacteria‐responsive nanocarriers are designed to release antimicrobials only in the presence of infection‐specific cues. This selective activation ensures drug release precisely at the site of infection, avoiding premature or indiscriminate release, and enhancing efficacy.
Guillermo Landa, Gracia Mendoza, Silvia Irusta, Manuel Arruebo   +3 more
wiley   +1 more source

Enhanced autophagic clearance of amyloid-β via histone deacetylase 6-mediated V-ATPase assembly and lysosomal acidification protects against Alzheimer’s disease in vitro and in vivo

Neural Regeneration Research
Recent studies have suggested that abnormal acidification of lysosomes induces autophagic accumulation of amyloid-β in neurons, which is a key step in senile plaque formation.
Zhimin Long, Chuanhua Ge, Yueyang Zhao, Yuanjie Liu, Qinghua Zeng, Qing Tang, Zhifang Dong, Guiqiong He   +7 more
doaj   +1 more source

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

Molecular 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, David S. Millington, Deeksha S. Bali   +2 more
doaj   +1 more source

Turning Unpredictable Biomolecule Adsorption to Controlled Corona Formation: Focus on Carbon Nanomaterials

Advanced Materials, EarlyView.
Controlling the protein corona formation onto carbon nanomaterials (CNMs) enhances their functionalities as platforms for cancer theranostics. Here, we reviewed the effects of the intrinsic and acquired properties of CNMs on protein corona formation, the consequent biological and toxicological outcomes, and the strategies to reshape corona formation ...
Yajuan Zou, Yalei Hu, Jie Yu, Naoki Komatsu, Yuta Nishina, Alberto Bianco   +5 more
wiley   +1 more source

A novel allosteric GCase modulator prevents Tau accumulation in GBA1 WT and GBA1 L444P/L444P cellular models

Scientific Reports
A slow decline in the autophagy-lysosomal pathway is a hallmark of the normal aging brain. Yet, an acceleration of this cellular function may propel neurodegenerative events. In fact, mutations in genes associated with the autophagy-lysosomal pathway can
Matteo Ciccaldo, Natàlia Pérez-Carmona, Ester Piovesana, Sara Cano-Crespo, Ana Ruano, Aida Delgado, Ilaria Fregno, Beatriz Calvo-Flores Guzmán, Manolo Bellotto, Maurizio Molinari, Joanne Taylor, Stéphanie Papin, Ana María García-Collazo, Paolo Paganetti   +13 more
doaj   +1 more source