SPECIFIC TARGETED ACTIVATION OF CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR)

摘要

This invention provides compositions and methods for enhancing CFTR expression in a cell. The invention also provides methods for treating cystic fibrosis. In one aspect, the invention provides methods for enhancing expression or cellular level of cystic fibrosis transmembrane conductance regulator (CFTR) in a cell. The methods entail contacting the cell with an agent that specifically disrupts or downregulates CFTR associated antisense long non-coding RNA (IncRNA) BG213071 and/or A1805947. Some of the methods are directed enhancing CFTR expression in an epithelial cell.