Repair of CFTR Folding Defects with Correctors that Function as Pharmacological Chaperones

2011 ◽  
pp. 23-37 ◽  
Author(s):  
Tip W. Loo ◽  
David M. Clarke
Keyword(s):  
Pharmacological Chaperones

scholarly journals Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases

2016 ◽  
Vol 52 (32) ◽  
pp. 5497-5515 ◽  
Author(s):  
Elena M. Sánchez-Fernández ◽  
José M. García Fernández ◽  
Carmen Ortiz Mellet
Keyword(s):  
Lysosomal Storage Disorders ◽  
Lysosomal Storage ◽  
Future Outlook ◽  
Gm1 Gangliosidosis ◽  
Pharmacological Chaperones ◽  
Storage Disorders

Recent advancements and future outlook on pharmacological chaperones for lysosomal storage disorders using glycomimetics are discussed.

Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR

2021 ◽  
Author(s):  
Nesrine Baatallah ◽  
Ahmad Elbahnsi ◽  
Jean-Paul Mornon ◽  
Benoit Chevalier ◽  
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...  
Keyword(s):  
Binding Sites ◽  
Pharmacological Chaperones ◽  
Domain Stability

Pharmacological Chaperones in Nephrogenic Diabetes Insipidus

BioDrugs ◽  
2007 ◽  
Vol 21 (3) ◽  
pp. 157-166 ◽  
Author(s):  
Joris H Robben ◽  
Peter M T Deen
Keyword(s):  
Diabetes Insipidus ◽  
Nephrogenic Diabetes Insipidus ◽  
Pharmacological Chaperones

scholarly journals A novel triple combination of pharmacological chaperones improves F508del-CFTR correction

2018 ◽  
Vol 8 (1) ◽  
Author(s):  
Graeme W. Carlile ◽  
Qi Yang ◽  
Elizabeth Matthes ◽  
Jie Liao ◽  
Stevo Radinovic ◽  
...  
Keyword(s):  
Triple Combination ◽  
Pharmacological Chaperones

scholarly journals Simultaneous Control of Endogenous and User-Defined Genetic Pathways Using Unique ecDHFR Pharmacological Chaperones

2020 ◽  
Vol 27 (5) ◽  
pp. 622-634.e6 ◽  
Author(s):  
Prerana Ramadurgum ◽  
DaNae R. Woodard ◽  
Steffi Daniel ◽  
Hui Peng ◽  
Prema L. Mallipeddi ◽  
...  
Keyword(s):  
Genetic Pathways ◽  
Pharmacological Chaperones ◽  
Simultaneous Control

scholarly journals Stereoselective Synthesis of C-2 Alkylated Trihydroxypiperidines: Novel Pharmacological Chaperones for Gaucher Disease

2019 ◽  
Vol 10 (4) ◽  
pp. 621-626 ◽  
Author(s):  
Francesca Clemente ◽  
Camilla Matassini ◽  
Andrea Goti ◽  
Amelia Morrone ◽  
Paolo Paoli ◽  
...  
Keyword(s):  
Gaucher Disease ◽  
Stereoselective Synthesis ◽  
Pharmacological Chaperones
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