Search Results - mae+lewis

2 Results Sort By:
Beta-sitosterol lipid nanoparticles for aerosolized gene editing in lung diseases
Background Cystic fibrosis (CF) is a life-threatening genetic disorder caused by mutations in the CFTR gene, leading to progressive lung dysfunction. A particularly challenging mutation, W1282X, introduces a premature stop codon that results in non-functional CFTR protein. Patients with this mutation are largely unresponsive to current small molecule...
Published: 12/4/2025   |   Inventor(s): Debadyuti Ghosh, Mae Lewis
Keywords(s):  
Category(s): Life sciences > Therapeutics > Gene editing, Life sciences > Therapeutics > Drug delivery
Stably aerosolized mRNA for lung delivery
Problem Nucleic acid therapy has the potential to treat genetic diseases that affect the lungs. Localized delivery of nucleic acid cargos allows for higher doses than are achievable with systemic delivery and avoids both toxicity and off-target effects. One reason nucleic acid therapeutics have not yet achieved clinical approval for delivery to the...
Published: 11/25/2025   |   Inventor(s): Debadyuti Ghosh, Mae Lewis
Keywords(s):  
Category(s): Life sciences > Therapeutics > Drug delivery