2058 miRNA manipulation to improve CFTR correction in cystic fibrosis
OBJECTIVES/SPECIFIC AIMS: CFTR is the mutant protein that causes cystic fibrosis (CF), a fatal respiratory diseases affecting 1 in 3500 children. CFTR modulators are small molecules that directly address mutant CFTR function. Improving correction of the F508del CFTR mutation (affecting 90% of CF pat...
| Main Authors: | , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Cambridge University Press
2018-06-01
|
| Series: | Journal of Clinical and Translational Science |
| Online Access: | https://www.cambridge.org/core/product/identifier/S2059866118000961/type/journal_article |