Methods and Products for Increasing CFTR Levels

uah p 16026

Docket: UAH-P-16026

Technology

Cystic Fibrosis (CF) is a life shortening genetic disease that severely damages the respiratory and digestive systems by causing normal mucus and enzyme secretions to become thick and sticky, blocking important passageways in the lungs, intestines, pancreas, and liver. CF is caused by a defect in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. CFTR controls the movement of water and salt in and out of the body's cells.

Over 1,000 different mutations in CFTR with varying levels of CFTR function are known. The most successfully treated CF patients have mutations in which the amount of partially functional CFTR proteins is still relatively high. However, some patients have CFTR mutations that result in little to no functional CFTR proteins available.

Researchers at UAH have developed methods and products for increasing both normal and mutant CFTR protein expression in a cell. With a greater quantity of CFTR protein available for at least some functionality, current CF treatments could be more successful, resulting in higher quality of life for CF patients. In a lab setting, these researchers have successfully targeted the CFTR gene and increased CFTR protein expression almost 200-fold in lung cell lines, including lines with CFTR mutations. This technology can permanently and stably increase CFTR production in cell lines, allowing for improved drug screening.

Applications

  • Pharmaceuticals
  • Biomedical research

Advantages

  • Increases CFTR expression in cells
  • Higher quantity of functional CFTR RNA works synergistically with drugs to alleviate CF symptoms

Status

  • State of Development: Proof of concept
  • Licensing Status: Available for licensing
  • Patent Status: Patent pending