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Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán

2015, Número 1

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Rev Invest Clin 2015; 67 (1)


Pharmacoperones: Targeting Therapeutics Toward Diseases Caused by Protein Misfolding

Ulloa-Aguirre A, Zariñán T, Conn PM

Idioma: Ingles.
Referencias bibliográficas: 11
Paginas: 15-19
Archivo PDF: 320.76 Kb.


RESUMEN

Sin resumen.


REFERENCIAS (EN ESTE ARTÍCULO)

  1. Conn PM , Ulloa-Aguirre A. Pharmacological chaperones for misfolded gonadotropin-releasing hormone receptors. Adv Pharmacol. 2011;62:109-41.

  2. Ulloa-Aguirre A, Janovick JA, Brothers SP, Conn PM. Pharmacologic rescue of conformationally-defective proteins: implications for the treatment of human disease. Traffic. 2004;5:821-37.

  3. Ulloa-Aguirre A, Zarinan T, Dias JA, Conn PM. Mutations in G protein-coupled receptors that impact receptor trafficking and reproductive function. Mol Cell Endocrinol. 2014;382:411-23.

  4. Ishii S, Chang HH, Kawasaki K, et al. Mutant alpha-galactosidase A enzymes identified in Fabry disease patients with residual enzyme activity: biochemical characterization and restoration of normal intracellular processing by 1-deoxygalactonojirimycin. Biochem J. 2007;406:285-95.

  5. Gautherot J, Durand-Schneider AM, Delautier D, et al. Effects of cellular, chemical, and pharmacological chaperones on the rescue of a trafficking-defective mutant of the ATP-binding cassette transporter proteins ABCB1/ABCB4. J Biol Chem. 2012;287:5070-8.

  6. Conn PM, Ulloa-Aguirre A, Ito J, Janovick JA. G protein-coupled receptor trafficking in health and disease: lessons learned to prepare for therapeutic mutant rescue in vivo. Pharmacol Rev. 2007;59:225-50.

  7. Bernier V, Morello JP, Zarruk A, et al. Pharmacologic chaperones as a potential treatment for X-linked nephrogenic diabetes insipidus. J Am Soc Nephrol. 2006;17:232-43.

  8. Conn PM, Ulloa-Aguirre A. Trafficking of G-protein-coupled receptors to the plasma membrane: insights for pharmacoperone drugs. Trends Endocrinol Metab. 2010;21:190-7.

  9. Janovick JA, Stewart MD, Jacob D, et al. Restoration of testis function in hypogonadotropic hypogonadal mice harboring a misfolded GnRHR mutant by pharmacoperone drug therapy. Proc Natl Acad Sci USA. 2013;110:21030-5.

  10. Conn PM, Smithson DC, Hodder PS, et al. Transitioning pharmacoperones to therapeutic use: in vivo proof-of-principle and design of high throughput screens. Pharmacol Res. 2014;83:38-51.

  11. Kapoor H, Koolwal A, Singh A. Ivacaftor: a novel mutation modulating drug. J Clin Diagn Res. 2014;8:SE01-5.




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