Implementation of Water‐Soluble Cyclodextrin‐Based Polymers in Biomedical Applications: How Far Are We?
Cyclodextrin‐based polymers can be prepared starting from the naturally occurring
monomers following green and low‐cost procedures. They can be selectively derivatized …
monomers following green and low‐cost procedures. They can be selectively derivatized …
Integration of cyclodextrins and associated toxicities: A roadmap for high quality biomedical applications
F Trotta, T Loftsson, RS Gaud, R Trivedi… - Carbohydrate Polymers, 2022 - Elsevier
Cyclodextrins are extensively employed in drug delivery systems like inclusion complexes,
metal-organic frameworks, functionalized or PEGylated conjugates, and other nanocarrier …
metal-organic frameworks, functionalized or PEGylated conjugates, and other nanocarrier …
The NCATS Pharmaceutical Collection: a 10-year update
HighlightsNPC repurposing screens discovered compounds with new clinical
indications.Systematic NPC screening data establishes rich drug activity …
indications.Systematic NPC screening data establishes rich drug activity …
Neurofilament light chain in cerebrospinal fluid as a novel biomarker in evaluating both clinical severity and therapeutic response in Niemann-Pick disease type C1
N Agrawal, NY Farhat, N Sinaii, AD Do, C Xiao… - Genetics in …, 2023 - Elsevier
Abstract Purpose Niemann-Pick disease type C1 (NPC1) is a neurodegenerative lysosomal
disorder caused by pathogenic variants in NPC1. Disease progression is monitored using …
disorder caused by pathogenic variants in NPC1. Disease progression is monitored using …
The complexity of a monogenic neurodegenerative disease: More than two decades of therapeutic driven research into Niemann-Pick type C disease
N Hammond, AB Munkacsi, SL Sturley - Biochimica et Biophysica Acta …, 2019 - Elsevier
Abstract Niemann-Pick type C (NP-C) disease is a rare and fatal neurodegenerative disease
typified by aberrations in intracellular lipid transport. Cholesterol and other lipids accumulate …
typified by aberrations in intracellular lipid transport. Cholesterol and other lipids accumulate …
Microglia activation in Niemann–Pick disease, type C1 is amendable to therapeutic intervention
A Cougnoux, RA Drummond, AL Collar… - Human molecular …, 2018 - academic.oup.com
Niemann–Pick disease, type C1 (NPC1) is a neurodegenerative disorder with limited
treatment options. NPC1 is associated with neuroinflammation; however, attempts to …
treatment options. NPC1 is associated with neuroinflammation; however, attempts to …
Polyrotaxane-based systemic delivery of β-cyclodextrins for potentiating therapeutic efficacy in a mouse model of Niemann-Pick type C disease
A Tamura, N Yui - Journal of controlled release, 2018 - Elsevier
Abstract Niemann-Pick type C (NPC) disease is a fatal metabolic disorder characterized by
the lysosomal accumulation of cholesterol. Although 2-hydroxypropyl β-cyclodextrin (HP-β …
the lysosomal accumulation of cholesterol. Although 2-hydroxypropyl β-cyclodextrin (HP-β …
Different solubilizing ability of cyclodextrin derivatives for cholesterol in Niemann–Pick disease type C treatment
Y Yamada, M Fukaura‐Nishizawa… - Clinical and …, 2023 - Wiley Online Library
Abstract Background Niemann–Pick disease type C (NPC) is a fatal neurodegenerative
disorder caused by abnormal intracellular cholesterol trafficking. Cyclodextrins (CDs), the …
disorder caused by abnormal intracellular cholesterol trafficking. Cyclodextrins (CDs), the …
Hydroxypropyl-beta and-gamma cyclodextrins rescue cholesterol accumulation in Niemann–Pick C1 mutant cell via lysosome-associated membrane protein 1
Abstract Niemann–Pick type C (NPC) disease is a fatal hereditary neurodegenerative
disorder characterized by a massive accumulation of cholesterol in lysosomes and late …
disorder characterized by a massive accumulation of cholesterol in lysosomes and late …
Insight into the role of extracellular vesicles in lysosomal storage disorders
B Tancini, S Buratta, K Sagini, E Costanzi, F Delo… - Genes, 2019 - mdpi.com
Extracellular vesicles (EVs) have received increasing attention over the last two decades.
Initially, they were considered as just a garbage disposal tool; however, it has progressively …
Initially, they were considered as just a garbage disposal tool; however, it has progressively …