Molecular therapy: liposome nanoparticles enhance gene therapy in the treatment of cystic fibrosis

June 26, 2018 / biogood / - although researchers began to use gene therapy to correct the cystic fibrosis transmembrane conductance regulator (CFTR) to treat cystic fibrosis a few years ago, the potential of gene therapy for cystic fibrosis has not been concerned Image source: Jerry Nick, M.D / Wikipedia's nanoparticle delivery of mRNA is a powerful technology for delivering genetic material to a large number of widely distributed cell populations, such as respiratory epithelial cells In order to study the clinical application prospect of nanoparticle delivery of mRNA in the treatment of cystic fibrosis, scientists from the Department of pharmacy of the school of medicine of Oregon State University and other units recently developed a kind of liposome based nanoparticle (LNP) based on clinical lipid, and used it to package and deliver chemically modified CFTR MRNA (cmcftr) entered the bronchial epithelial cells from patients It was found that the number of cmcftr was increased and its main function as chloride channel was restored The related research results were recently published in molecular therapy, entitled "lipid nanoparticle delivered chemically modified mRNA restores chloride secret in cyclic fibrosis" In addition, the researchers found that lnp-cmcftr inhalation through the nasal cavity can restore the CFTR mediated secretion of chloride ions into the respiratory epithelium of CFTR knockout mice, and maintain the function for up to 14 days The researchers found that after three days of transfection, the activity of CFTR reached its peak, and it recovered 55% of the function of chloride net circulation The intensity of this reaction is stronger than that of liposome delivery of CFTR DNA, and the clinical effect is equivalent to that of ivakato Therefore, lnp-cmrna system represents a powerful technical platform for the treatment of cystic fibrosis and other single gene mutation diseases Original source: EMA Robinson et al, lipid nanoparticle delivered chemically modified mRNA restores chloride secret in cyclic fibrosis, molecular therapy (2018) Doi: 10.1016/j.ymthe.2018.05.014