Amaral, Margarida D. and Kunzelmann, Karl (2007) Molecular targeting of CFTR as a therapeutic approach to cystic fibrosis. TRENDS IN PHARMACOLOGICAL SCIENCES, 28 (7). pp. 334-341. ISSN 0165-6147
Full text not available from this repository. (Request a copy)Abstract
One of the major challenges facing the pharmaceutical field is the identification of novel, 'druggable' targets common to distinct diseases that, despite their clinical diversity, share the same basic molecular defect(s) - thus, being termed 'horizontal diseases'. Membrane proteins constitute one of the largest families in the human genome and, given their major roles in cells and organisms, they are relevant to common human disorders such as cardiovascular disease and cancer, but also to rare genetic conditions such as cystic fibrosis (CF). Here, we review therapeutic approaches to correcting the basic defect in CF,which is caused mainly by the intracellular retention of a misfolded protein, and focus on various recent drug-discovery strategies for this important and paradigmatic disease. These strategies have possible applications in many membrane protein disorders, including other channelopathies. The mechanisms of action of potent and specific compounds, representing promising drug leads for CF pharmacotherapy, are explained and discussed.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | TRANSMEMBRANE CONDUCTANCE REGULATOR; NUCLEOTIDE-BINDING DOMAIN; AIRWAY EPITHELIAL-CELLS; CHLORIDE-CHANNEL; PHARMACOLOGICAL CHAPERONES; TRANSPORT DEFECT; DELTA-F508 CFTR; DOWN-REGULATION; RESCUE; CORRECTORS; |
| Subjects: | 500 Science > 570 Life sciences |
| Divisions: | Biology, Preclinical Medicine > Institut für Physiologie > Prof. Dr. Karl Kunzelmann |
| Depositing User: | Petra Gürster |
| Date Deposited: | 30 Oct 2020 09:15 |
| Last Modified: | 30 Oct 2020 09:15 |
| URI: | https://pred.uni-regensburg.de/id/eprint/32587 |
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