FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W4220947609> ?p ?o ?g. }

• W4220947609 endingPage "400" @default.
• W4220947609 startingPage "389" @default.
• W4220947609 abstract "Cyclodextrins (CDs) are used not only as pharmaceutical excipients but also as active pharmaceutical ingredients. CDs can act as artificial carriers or shuttles to ameliorate lipid transport disorders. Niemann-Pick disease type C (NPC) is an inherited, progressive neurodegenerative disorder caused by mutations in NPC1 or NPC2 genes, in which unesterified cholesterol accumulates in lysosomes and the transport of cholesterol from lysosomes to the endoplasmic reticulum is impaired. 2-Hydroxypropyl-β-CD (HPBCD) has activity as a cholesterol shuttle and can attenuate NPC-related manifestations in model cells and animals. HPBCD can also be an effective treatment for NPC patients, but has produced lung damage and ototoxicity at therapeutic doses in clinical trials. Like HPBCD, 2-hydroxypropyl-γ-CD (HPGCD) can normalize disrupted cholesterol homeostasis in cells derived from NPC patients and NPC model mice. HPGCD interacts with unesterified cholesterol with a mode of interaction distinct from that of HPBCD and acts as a fine-tuned cholesterol shuttle for the treatment of NPC with a wider safety margin than HPBCD in terms of ototoxicity and pulmonary toxicity. By bridging clinical and basic research, it is hoped that progress will be made in the development of therapeutic agents against neurodegenerative lipid storage disorders that share common pathogenic mechanisms with NPC." @default.
• W4220947609 created "2022-04-03" @default.
• W4220947609 creator A5035850424 @default.
• W4220947609 date "2022-04-01" @default.
• W4220947609 modified "2023-10-18" @default.
• W4220947609 title "Inclusion Solves Insolubility —Translational Research Cycle from Bedside to Bench and Bench to Bedside for Drug Development Targeting Niemann-Pick Disease Type C—" @default.
• W4220947609 cites W1481040247 @default.
• W4220947609 cites W1552108885 @default.
• W4220947609 cites W1568412294 @default.
• W4220947609 cites W1865630485 @default.
• W4220947609 cites W1892965049 @default.
• W4220947609 cites W1901815064 @default.
• W4220947609 cites W1902906608 @default.
• W4220947609 cites W1968382333 @default.
• W4220947609 cites W1994735062 @default.
• W4220947609 cites W1994886732 @default.
• W4220947609 cites W1996481160 @default.
• W4220947609 cites W1999925370 @default.
• W4220947609 cites W2002646402 @default.
• W4220947609 cites W2013273719 @default.
• W4220947609 cites W2020458107 @default.
• W4220947609 cites W2021248130 @default.
• W4220947609 cites W2031036779 @default.
• W4220947609 cites W2043457913 @default.
• W4220947609 cites W2055091943 @default.
• W4220947609 cites W2055806250 @default.
• W4220947609 cites W2057099934 @default.
• W4220947609 cites W2061661225 @default.
• W4220947609 cites W2065408118 @default.
• W4220947609 cites W2067882022 @default.
• W4220947609 cites W2068262899 @default.
• W4220947609 cites W2073749205 @default.
• W4220947609 cites W2085578246 @default.
• W4220947609 cites W2094205187 @default.
• W4220947609 cites W2101383059 @default.
• W4220947609 cites W2116144339 @default.
• W4220947609 cites W2117321286 @default.
• W4220947609 cites W2136116905 @default.
• W4220947609 cites W2145267271 @default.
• W4220947609 cites W2147641578 @default.
• W4220947609 cites W2162400220 @default.
• W4220947609 cites W2344443137 @default.
• W4220947609 cites W2589097599 @default.
• W4220947609 cites W2711429970 @default.
• W4220947609 cites W2744065025 @default.
• W4220947609 cites W2767267840 @default.
• W4220947609 cites W2767552310 @default.
• W4220947609 cites W2790546302 @default.
• W4220947609 cites W2920125937 @default.
• W4220947609 cites W2945280958 @default.
• W4220947609 cites W3004336560 @default.
• W4220947609 cites W3120886202 @default.
• W4220947609 cites W3145762250 @default.
• W4220947609 cites W3194097199 @default.
• W4220947609 cites W4321429263 @default.
• W4220947609 doi "https://doi.org/10.1248/yakushi.21-00215" @default.
• W4220947609 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/35370195" @default.
• W4220947609 hasPublicationYear "2022" @default.
• W4220947609 type Work @default.
• W4220947609 citedByCount "1" @default.
• W4220947609 countsByYear W42209476092022 @default.
• W4220947609 crossrefType "journal-article" @default.
• W4220947609 hasAuthorship W4220947609A5035850424 @default.
• W4220947609 hasBestOaLocation W42209476091 @default.
• W4220947609 hasConcept C126322002 @default.
• W4220947609 hasConcept C158617107 @default.
• W4220947609 hasConcept C185592680 @default.
• W4220947609 hasConcept C19527891 @default.
• W4220947609 hasConcept C2778163477 @default.
• W4220947609 hasConcept C2779489717 @default.
• W4220947609 hasConcept C2780035454 @default.
• W4220947609 hasConcept C2780635549 @default.
• W4220947609 hasConcept C3020000666 @default.
• W4220947609 hasConcept C55493867 @default.
• W4220947609 hasConcept C71924100 @default.
• W4220947609 hasConcept C98274493 @default.
• W4220947609 hasConceptScore W4220947609C126322002 @default.
• W4220947609 hasConceptScore W4220947609C158617107 @default.
• W4220947609 hasConceptScore W4220947609C185592680 @default.
• W4220947609 hasConceptScore W4220947609C19527891 @default.
• W4220947609 hasConceptScore W4220947609C2778163477 @default.
• W4220947609 hasConceptScore W4220947609C2779489717 @default.
• W4220947609 hasConceptScore W4220947609C2780035454 @default.
• W4220947609 hasConceptScore W4220947609C2780635549 @default.
• W4220947609 hasConceptScore W4220947609C3020000666 @default.
• W4220947609 hasConceptScore W4220947609C55493867 @default.
• W4220947609 hasConceptScore W4220947609C71924100 @default.
• W4220947609 hasConceptScore W4220947609C98274493 @default.
• W4220947609 hasIssue "4" @default.
• W4220947609 hasLocation W42209476091 @default.
• W4220947609 hasLocation W42209476092 @default.
• W4220947609 hasOpenAccess W4220947609 @default.
• W4220947609 hasPrimaryLocation W42209476091 @default.
• W4220947609 hasRelatedWork W1690219722 @default.
• W4220947609 hasRelatedWork W1967516512 @default.
• W4220947609 hasRelatedWork W1984779508 @default.
• W4220947609 hasRelatedWork W1991766132 @default.
• W4220947609 hasRelatedWork W2053942928 @default.

• next