POLYMER-NUCLEIC ACID NANOPARTICLES FOR GENE EDITING
Provided are nanoparticles comprising a biodegradable synthetic polymer, one or more nucleic acids, and a stabilizing agent, such as PEGylated lipid and their use for delivering genes to the lung epithelium and other tissues for gene editing to correct genetic mutations in diseases, such as cystic fibrosis.
Fecha publicación: 2024/05/10
Número: WO2024098053A1 (A1)
Solicitante: THE JOHNS HOPKINS UNIV?[US]
Inventor: GREEN JORDAN J?[US]; TZENG STEPHANY YI?[US] (2)