Results 81 to 90 of about 477,265 (189)

MOESM2 of A pharmacological chaperone improves memory by reducing Aβ and tau neuropathology in a mouse model with plaques and tangles

, 2020
Additional file 2: Figure S2. Pharmacological chaperone affects retromer complex levels in hippocampi of 3xTg mice. (A) Representative western blot analysis of VPS35, VPS26b and VPS29 proteins in hippocampus homogenates from 3xTg mice treated with TPT or
Benjamin Blass (8342454), Jin Chiu (8342448), Domenico Praticò (336367), Jian-Guo Li (2448088), Mercy Ramanjulu (8342451)   +4 more
core   +1 more source

Therapeutic targeting of cellular prion protein: toward the development of dual mechanism anti-prion compounds

Neural Regeneration Research
PrPSc, a misfolded, aggregation-prone isoform of the cellular prion protein (PrPC), is the infectious prion agent responsible for fatal neurodegenerative diseases of humans and other mammals.
Antonio Masone, Chiara Zucchelli, Enrico Caruso, Giovanna Musco, Roberto Chiesa   +4 more
doaj   +1 more source

5CBranched Deoxynojirimycin: Strategy for Designing a 1Deoxynojirimycin-Based Pharmacological Chaperone with a Nanomolar Affinity for Pompe Disease

, 2022
In recent years, the function of pharmacological chaperones as a “thermodynamic stabilizer” has been attracting attention in combination therapy. The coadministration of a pharmacological chaperone and recombinant human acid α-glucosidase (rhGAA) leads ...
Robert J. Nash, Kenta Shinzawa (11992858), Izumi Nakagome, Atsushi Kato (264067), Yi-Xian Li (1600036), Nobutada Tanaka, Kosuke Yoshimura (11992852), Nobutada Tanaka (1720114), Atsushi Kato, Chu-Yi Yu, Tian-Tian Lu, Uta Kanekiyo, Yue-Mei Jia, Yue-Mei Jia (1600030), Mana Kishida (11992855), George W. J. Fleet (9017531), Robert J. Nash (701546), Chu-Yi Yu (1357497), Mana Kishida, Tian-Tian Lu (11992849), Kenta Shinzawa, Yi-Xian Li, Tomoki Yoshida (5368634), Kosuke Yoshimura, Izumi Nakagome (1608361), Tomoki Yoshida, Uta Kanekiyo (11992846), George W. J. Fleet   +27 more
core   +1 more source

Auxiliary protein and chaperone regulation of neuronal nicotinic receptor subtype expression and function

Pharmacological Research
Neuronal nicotinic acetylcholine receptors (nAChRs) are a family of pentameric, ligand-gated ion channels that are located on the surface of neurons and non-neuronal cells and have multiple physiological and pathophysiological functions.
Cecilia Gotti, Francesco Clementi, Michele Zoli   +2 more
doaj   +1 more source

Pharmacological chaperone therapy for Gaucher disease: a patent review [PDF]

Expert Opinion on Therapeutic Patents, 2011
Mutations in the gene encoding for acid β-glucosidase (β-glucocerebrosidase, GlcCerase) are seen in Gaucher disease (GD), which give rise to significant protein misfolding effects and result in progressive accumulation of glucosyl ceramide. The main treatment for GD is enzyme replacement therapy (ERT). The iminosugar glycosidase inhibitor N-(n-butyl)-1-
Benito, Juan Manuel, García Fernández, José Manuel, Ortiz Mellet, Carmen   +2 more
openaire   +5 more sources

Structural basis of pharmacological chaperoning for human β-galactosidase [PDF]

Acta Crystallographica Section A Foundations and Advances, 2014
GM1-gangliosidosis and Morquio B are rare lysosomal storage diseases associated with a neurodegenerative disorder or dwarfism and skeletal abnormalities, respectively. These diseases are caused by deficiencies in the lysosomal enzyme human β-Galactosidase (β-Gal), frequently related to misfolding and subsequent endoplasmic reticulum-associated ...
Suzuki, Hironori, Ohto, Umeharu, Higaki, Katsumi, Mena Barragán, Teresa, Aguilar Moncayo, Matilde, Ortiz Mellet, Carmen, Nanba, Eiji, García Fernández, José Manuel, Suzuki, Yoshiyuki, Shimizu, Toshiyuki   +9 more
openaire   +6 more sources

Global Landscape of SLC37A4 Variants and Their Potential Amenability to Pharmacological Chaperone Therapy in Glycogen Storage Disease Type Ib

Sci
Glycogen storage disease Ib (GSD Ib) is an ultra-rare metabolic disease caused by variants in the SLC37A4 gene affecting activity of the glucose-6-phosphate transporter (G6PT).
Anita Skakic, Kristel Klaassen, Marina Andjelkovic, Jovana Komazec, Marina Parezanovic, Nikola Jocic, Maja Djordjevic Milosevic, Maja Stojiljkovic   +7 more
doaj   +1 more source

Effects of YM087 and VPA985 on the T237M mutant receptor functionality in nephrogenic diabetes insipidus

Türk Biyokimya Dergisi
Mutations detected in the AVPR2 gene (arginine vasopressin type 2 receptor) are known to cause nephrogenic diabetes insipidus (NDI). Several pharmacological chaperones (PCs) target misfolded AVPR2 proteins and rescue them from the quality control system ...
Avcu Elif Merve, Erdem Tuncdemir Beril, Saglar Ozer Emel   +2 more
doaj   +1 more source

Image_12_Structure-Activity Relationships of Dopamine Transporter Pharmacological Chaperones.TIF

, 2022
Mutations in the dopamine transporter gene (SLC6A3) have been implicated in many human diseases. Among these is the infantile parkinsonism-dystonia known as Dopamine Transporter Deficiency Syndrome (DTDS).
Deborah C. Mash (12516796), Ali Salahpour (239589), Charles Sutton (5191816), Dong Han (199240), Pieter Beerepoot (5712680), Amy J. Ramsey (2002120), Bruce Blough (2042590), David E. Olson (1987003), Erin Q. Williams (12516787), Reza Nazari (12516793), Hoomam Homsi (12516790)   +10 more
core   +1 more source

Crystal structure of the P pilus rod subunit PapA [PDF]

, 2007
P pili are important adhesive fibres involved in kidney infection by uropathogenic Escherichia coli strains. P pili are assembled by the conserved chaperone-usher pathway, which involves the PapD chaperone and the PapC usher.
Bullitt, E, Hultgren, SJ, Hultgren, Scott J, Denis Verger, Waksman, Gabriel, Hultgren, Scott J., Verger, Denis, Esther Bullitt, Scott J Hultgren, Bullitt, Esther, Waksman, G, Verger, D, Gabriel Waksman, Verger Denis, Waksman Gabriel, Bullitt Esther, Hultgren Scott J   +16 more
core   +1 more source