Literature DB >> 14685252

Therapeutic approaches to protein-misfolding diseases.

Fred E Cohen1, Jeffery W Kelly.   

Abstract

Several sporadic and genetic diseases are caused by protein misfolding. These include cystic fibrosis and other devastating diseases of childhood as well as Alzheimer's, Parkinson's and other debilitating maladies of the elderly. A unified view of the molecular and cellular pathogenesis of these conditions has led to the search for chemical chaperones that can slow, arrest or revert disease progression. Molecules are now emerging that link our biophysical insights with our therapeutic aspirations.

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Year:  2003        PMID: 14685252     DOI: 10.1038/nature02265

Source DB:  PubMed          Journal:  Nature        ISSN: 0028-0836            Impact factor:   49.962


  250 in total

1.  Altered trafficking and stability of polycystins underlie polycystic kidney disease.

Authors:  Yiqiang Cai; Sorin V Fedeles; Ke Dong; Georgia Anyatonwu; Tamehito Onoe; Michihiro Mitobe; Jian-Dong Gao; Dayne Okuhara; Xin Tian; Anna-Rachel Gallagher; Zhangui Tang; Xiaoli Xie; Maria D Lalioti; Ann-Hwee Lee; Barbara E Ehrlich; Stefan Somlo
Journal:  J Clin Invest       Date:  2014-11-03       Impact factor: 14.808

Review 2.  Pharmacoperones: a new therapeutic approach for diseases caused by misfolded G protein-coupled receptors.

Authors:  Alfredo Ulloa-Aguirre; P Michael Conn
Journal:  Recent Pat Endocr Metab Immune Drug Discov       Date:  2011-01

3.  A missense LAMB2 mutation causes congenital nephrotic syndrome by impairing laminin secretion.

Authors:  Ying Maggie Chen; Yamato Kikkawa; Jeffrey H Miner
Journal:  J Am Soc Nephrol       Date:  2011-04-21       Impact factor: 10.121

4.  Insulin forms amyloid in a strain-dependent manner: an FT-IR spectroscopic study.

Authors:  Wojciech Dzwolak; Vytautas Smirnovas; Ralf Jansen; Roland Winter
Journal:  Protein Sci       Date:  2004-05-28       Impact factor: 6.725

Review 5.  Heat shock transcription factor 1 as a therapeutic target in neurodegenerative diseases.

Authors:  Daniel W Neef; Alex M Jaeger; Dennis J Thiele
Journal:  Nat Rev Drug Discov       Date:  2011-12-01       Impact factor: 84.694

6.  Mechanistic studies of substrate-assisted inhibition of ubiquitin-activating enzyme by adenosine sulfamate analogues.

Authors:  Jesse J Chen; Christopher A Tsu; James M Gavin; Michael A Milhollen; Frank J Bruzzese; William D Mallender; Michael D Sintchak; Nancy J Bump; Xiaofeng Yang; Jingya Ma; Huay-Keng Loke; Qing Xu; Ping Li; Neil F Bence; James E Brownell; Lawrence R Dick
Journal:  J Biol Chem       Date:  2011-10-03       Impact factor: 5.157

7.  The C-terminal repeating units of CsgB direct bacterial functional amyloid nucleation.

Authors:  Neal D Hammer; Bryan A McGuffie; Yizhou Zhou; Matthew P Badtke; Ashley A Reinke; Kristoffer Brännström; Jason E Gestwicki; Anders Olofsson; Fredrik Almqvist; Matthew R Chapman
Journal:  J Mol Biol       Date:  2012-06-07       Impact factor: 5.469

8.  Improving binding specificity of pharmacological chaperones that target mutant superoxide dismutase-1 linked to familial amyotrophic lateral sclerosis using computational methods.

Authors:  Richard J Nowak; Gregory D Cuny; Sungwoon Choi; Peter T Lansbury; Soumya S Ray
Journal:  J Med Chem       Date:  2010-04-08       Impact factor: 7.446

9.  Discovery and characterization of a mammalian amyloid disaggregation activity.

Authors:  Amber N Murray; James P Solomon; Ya-Juan Wang; William E Balch; Jeffery W Kelly
Journal:  Protein Sci       Date:  2010-04       Impact factor: 6.725

10.  Pharmacologic inhibition of N-linked glycan trimming with kifunensine disrupts GLUT1 trafficking and glucose uptake.

Authors:  Evans K Lodge; Jedediah D Bell; Emily M Roloff; Kathryn E Hamilton; Larry L Louters; Brendan D Looyenga
Journal:  Biochimie       Date:  2020-04-13       Impact factor: 4.079

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