Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis
| Autor: | Elena K Schneider-Futschik |
|---|---|
| Rok vydání: | 2019 |
| Předmět: |
0301 basic medicine
congenital hereditary and neonatal diseases and abnormalities Genetic enhancement Gene delivery Biology Cystic fibrosis Ivacaftor 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine Genome editing Genetics medicine CRISPR Molecular Biology Lumacaftor respiratory system medicine.disease Cystic fibrosis transmembrane conductance regulator respiratory tract diseases 030104 developmental biology chemistry 030220 oncology & carcinogenesis Cancer research biology.protein Molecular Medicine medicine.drug |
| Zdroj: | Gene Therapy. 26:354-362 |
| ISSN: | 1476-5462 0969-7128 |
| Popis: | Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent advent of the FDA-approved CFTR modulator drug ivacaftor, alone or in combination with lumacaftor or tezacaftor, has enabled treatment of the majority of patients suffering from CF. Even before the identification of the CFTR gene, gene therapy was put forward as a viable treatment option for this genetic condition. However, initial enthusiasm has been hampered as CFTR gene delivery to the lungs has proven to be more challenging than expected. This review covers the contemporary clinical and scientific knowledge base for small molecule CFTR modulator drug therapy, gene delivery vectors and CRISPR/Cas9 gene editing and highlights the prospect of these technologies for future treatment options. |
| Databáze: | OpenAIRE |
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