Strategies in early clinical development for the treatment of basic defects of cystic fibrosis
| Autor: | Sabrina Noël, Barbara Dhooghe, Jeremy B. Haaf, Teresinha Leal |
|---|---|
| Rok vydání: | 2016 |
| Předmět: |
0301 basic medicine
Cystic Fibrosis Transcription Genetic Genetic enhancement Cystic Fibrosis Transmembrane Conductance Regulator Disease Bioinformatics Cystic fibrosis Ivacaftor 03 medical and health sciences chemistry.chemical_compound Animals Humans Medicine Pharmacology (medical) Pharmacology business.industry Lumacaftor General Medicine Potentiator medicine.disease Ataluren 030104 developmental biology chemistry Mutation Protein repair business medicine.drug |
| Zdroj: | Expert Opinion on Investigational Drugs. 25:423-436 |
| ISSN: | 1744-7658 1354-3784 |
| Popis: | Twenty-six years after the identification of the gene responsible for cystic fibrosis (CF), controversies still surround the pathogenesis of the disease that continues to burden and shorten lives. Therefore, finding effective therapeutic strategies that target the basic defect of CF is crucially needed.This review offers a comprehensive survey of fundamental therapies in early stages of development for the treatment of CF. The first part describes recent strategies targeting the basic defect either at the gene or at the transcript level. The second part summarizes a panel of novel strategies targeting protein repair. The third part reports strategies targeting non-CFTR channels.Recent major breakthroughs in CF therapy have been made, raising hope to find a cure for CF. Apart from Vertex corrector and potentiator molecules (lumacaftor, ivacaftor, VX-661) and from ataluren, used to correct nonsense mutations, most compounds being currently tested are in very early (I-II) phases of development and definitive clinical results are keenly expected. Among the broad list of molecules and strategies being tested, the QR-010 compound and inhibitors of phosphodiesterase type 5 (sildenafil, vardenafil) could reveal a strong potentiality as therapeutic candidates to cure CF. |
| Databáze: | OpenAIRE |
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