Pharmacological Chaperones: Beyond Conformational Disorders

2017 ◽  
pp. 135-153 ◽  
Author(s):  
Nancy J. Leidenheimer
Keyword(s):  
Pharmacological Chaperones ◽  
Conformational Disorders

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 ◽  
Iwona Pranke ◽  
...  
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

Protein Quality Control in Neurodegeneration and Neuroprotection

2020 ◽  
pp. 1-24
Author(s):  
Yasmeena Akhter ◽  
Jahangir Nabi ◽  
Hinna Hamid ◽  
Nahida Tabassum ◽  
Faheem Hyder Pottoo ◽  
...  
Keyword(s):  
Quality Control ◽  
Neurodegenerative Disorders ◽  
Protein Quality ◽  
Protein Quality Control ◽  
Ubiquitin Proteasome System ◽  
Security System ◽  
Conformational Disorders ◽  
Ubiquitin Proteasome ◽  
Multi Level

Proteostasis is essential for regulating the integrity of the proteome. Disruption of proteostasis under some rigorous conditions leads to the aggregation and accumulation of misfolded toxic proteins, which plays a central role in the pathogenesis of protein conformational disorders. The protein quality control (PQC) system serves as a multi-level security system to shield cells from abnormal proteins. The intrinsic PQC systems maintaining proteostasis include the ubiquitin-proteasome system (UPS), chaperon-mediated autophagy (CMA), and autophagy-lysosome pathway (ALP) that serve to target misfolded proteins for unfolding, refolding, or degradation. Alterations of PQC systems in neurons have been implicated in the pathogenesis of various neurodegenerative disorders. This chapter provides an overview of PQC pathways to set a framework for discussion of the role of PQC in neurodegenerative disorders. Additionally, various pharmacological approaches targeting PQC are summarized.

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