Results 71 to 80 of about 477,265 (189)
Ambroxol: A potential therapeutics against neurodegeneration
Health Sciences Review, 2023 Ambroxol hydrochloride is a pharmacological chaperone approved by FDA as an expectorant. Currently, it is used as an antitussive and anti-asthmatic agent.
Prajwal Dhanve +4 more
doaj +1 more source
Wrapping the alpha-crystallin domain fold in a chaperone assembly [PDF]
, 2005 Small heat shock proteins (sHsps) are oligomers that perform a protective function by binding denatured proteins. Although ubiquitous, they are of variable sequence except for a C-terminal similar to 90-residue "alpha-crystallin domain".
Kappé, G. +9 more
core +1 more source
Carnitine is a pharmacological allosteric chaperone of the human lysosomal α-glucosidase
, 2022 Pompe disease is an inherited metabolic disorder due to the deficiency of the lysosomal acid α-glucosidase (GAA). The only approved treatment is enzyme replacement therapy with the recombinant enzyme (rhGAA).
Roberta Iacono (4610482) +12 more
core +1 more source
Investigation of novel pharmacological chaperones for Gaucher Disease
Journal of Molecular Graphics and Modelling, 2017 Beta-Glucocerebrosidase (GBA) is a lysosomal protein that is responsible for the hydrolysis of glycosylceramide into glucose and ceramide. Mutations in GBA lead to the accumulation of glycosylceramide in the lysosome causing an enlargement of the spleen and the liver and skeletal deformations. This disease is called Gaucher Disease.
Yilmazer, Buge +5 more
openaire +3 more sources
Fundamental ResearchProtein misfolding and aggregation are crucial pathogenic factors for cataracts, which are the leading cause of visual impairment worldwide. α-crystallin, as a small molecular chaperone, is involved in preventing protein misfolding and maintaining lens ...
Huaxia Wang +7 more
doaj +1 more source
Frontiers in Cellular and Infection Microbiology, 2016 Brucella has been reported to impair placental trophoblasts, a cellular target where Brucella efficiently replicates in association with the endoplasmic reticulum (ER), and ultimately trigger abortion in pregnant animals.
Xiangguo eWang +9 more
doaj +1 more source
Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
New England Journal of Medicine, 2016 Fabry's disease, an X-linked disorder of lysosomal α-galactosidase deficiency, leads to substrate accumulation in multiple organs. Migalastat, an oral pharmacologic chaperone, stabilizes specific mutant forms of α-galactosidase, increasing enzyme trafficking to lysosomes.The initial assay of mutant α-galactosidase forms that we used to categorize 67 ...
Germain, Dominique P +39 more
openaire +8 more sources
Hsp70- and Hsp90-mediated proteasomal degradation underlies TPIsugarkill pathogenesis in Drosophila
Neurobiology of Disease, 2010 Triosephosphate isomerase (TPI) deficiency is a severe glycolytic enzymopathy that causes progressive locomotor impairment and neurodegeneration, susceptibility to infection, and premature death. The recessive missense TPIsugarkill mutation in Drosophila
Stacy L. Hrizo, Michael J. Palladino
doaj +1 more source
Novel pharmacological chaperones that correct phenylketonuria in mice [PDF]
Human Molecular Genetics, 2012 Phenylketonuria (PKU) is caused by inherited phenylalanine-hydroxylase (PAH) deficiency and, in many genotypes, it is associated with protein misfolding. The natural cofactor of PAH, tetrahydrobiopterin (BH(4)), can act as a pharmacological chaperone (PC) that rescues enzyme function.
Sandra, Santos-Sierra +10 more
openaire +2 more sources
, 2016 Exploiting “chaperone molecule” to navigate the successful assembly energy landscapes has been extensively used in biological systems, whereas in artifical supramolecular systems the “chaperone-assisted” assembly strategy to be used for the synthesis of ...
Jingwei Ji (2616013) +7 more
core +1 more source