Tuning protein folding in lysosomal storage diseases: the chemistry behind pharmacological chaperones

被引：69

作者：

Pereira, David M. ^{[1
]}

Valentao, Patricia ^{[1
]}

Andrade, Paula B. ^{[1
]}

机构：

[1] Univ Porto, REQUIMTE LAQV, Lab Farmacognosia, Dept Quim,Fac Farm, Rua Jorge Viterbo Ferreira 228, P-4050313 Oporto, Portugal

来源：

CHEMICAL SCIENCE | 2018年 / 9卷 / 07期

关键词：

TYPE-1; GAUCHER-DISEASE; GLOBOID-CELL LEUKODYSTROPHY; ENZYME REPLACEMENT THERAPY; ACID-BETA-GLUCOSIDASE; KRABBE-DISEASE; CHEMICAL CHAPERONES; ALPHA-GALACTOSIDASE; MOLECULAR-BASIS; FABRY DISEASE; ENDOPLASMIC-RETICULUM;

D O I：

10.1039/c7sc04712f

中图分类号：

O6 [化学];

学科分类号：

0703 ;

摘要：

Misfolding of proteins is the basis of several proteinopathies. Chemical and pharmacological chaperones are small molecules capable of inducing the correct conformation of proteins, thus being of interest for human therapeutics. The most recent developments in medicinal chemistry and in the drug development of pharmacological chaperones are discussed, with focus on lysosomal storage diseases.

引用

页码：1740 / 1752

页数：13

共 35 条

[21] Cyclodextrin-Based Iminosugar Click Clusters: The First Examples of Multivalent Pharmacological Chaperones for the Treatment of Lysosomal Storage Disorders
Decroocq, Camille
Rodriguez-Lucena, David
Ikeda, Kyoko
Asano, Naoki
Compain, Philippe
CHEMBIOCHEM, 2012, 13 (05) : 661 - 664
[22] Protein Aggregation and Dysfunction of Autophagy-Lysosomal Pathway: A Vicious Cycle in Lysosomal Storage Diseases
Monaco, Antonio
Fraldi, Alessandro
FRONTIERS IN MOLECULAR NEUROSCIENCE, 2020, 13
[23] On the Design of Broad Based Screening Assays to Identify Potential Pharmacological Chaperones of Protein Misfolding Diseases
Naik, Subhashchandra
Zhang, Na
Gao, Phillip
Fisher, Mark T.
CURRENT TOPICS IN MEDICINAL CHEMISTRY, 2012, 12 (22) : 2504 - 2522
[24] GENETIC OF PHARMACOLOGICAL LYSOSOMAL STORAGE DISEASES - EFFECTS OF GLYCOSAMINOGLYCANS AND SPHINGOLIPIDS PATHOLOGY OF DENTAL-TISSUES
GOLDBERG, M
GRITLI, A
BLOCHZUPAN, A
SEPTIER, D
LECOLLE, S
LEGRAND, JM
RUCH, JV
COMPTES RENDUS DES SEANCES DE LA SOCIETE DE BIOLOGIE ET DE SES FILIALES, 1993, 187 (05): : 596 - 607
[25] Pharmacological chaperone therapy for lysosomal storage disorders - leveraging aspects of the folding pathway to maximize activity of misfolded mutant proteins
Fan, Jian-Qiang
FEBS JOURNAL, 2007, 274 (19) : 4943 - 4943
[26] 4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations
Front, Sophie
Almeida, Sofia
Zoete, Vincent
Charollais-Thoenig, Julie
Gallienne, Estelle
Marmy, Celine
Pilloud, Vincent
Marti, Roger
Wood, Tim
Martin, Olivier R.
Demotz, Stephane
BIOORGANIC & MEDICINAL CHEMISTRY, 2018, 26 (20) : 5462 - 5469
[27] Pharmacological Regulation of Transgene Expression via Chemically Induced Dimerization for Treatment of Neuropathic Lysosomal Storage Diseases
Lin, Yi
Wang, Xiaohong
Rose, Kevin
Boateng-Antwi, Michael
Pan, Dao
MOLECULAR THERAPY, 2018, 26 (05) : 113 - 114
[28] Protein structural features predict responsiveness to pharmacological chaperone treatment for three lysosomal storage disorders
Woodard, Jaie
Zheng, Wei
Zhang, Yang
PLOS COMPUTATIONAL BIOLOGY, 2021, 17 (09)
[29] Murine Models of Lysosomal Storage Diseases Exhibit Differences in Brain Protein Aggregation and Neuroinflammation
Clarke, Jennifer
Kayatekin, Can
Viel, Catherine
Shihabuddin, Lamya
Sardi, Sergio Pablo
BIOMEDICINES, 2021, 9 (05)
[30] Protein dynamics, folding and misfolding: from basic physical chemistry to human conformational diseases
Ferreira, ST
De Felice, FG
FEBS LETTERS, 2001, 498 (2-3) : 129 - 134

← 1 2 3 4 →