LAUSR

HighlightsGene editing strategies for gene therapy of cystic fibrosis.Chemically modified mRNA for CFTR protein replacement therapy.Better viral and non‐viral vectors targeted to lung epithelium.Human and animal models for CF. &NA; Gene therapy for cystic fibrosis (CF) has been the subject of intense research over the last twenty‐five years or more, using both viral and liposomal delivery methods, but so far without the emergence of a clinical therapy. New approaches to CF gene therapy involving recent improvements to vector systems, both viral and non‐viral, as well as new nucleic acid technologies have led to renewed interest in the field. The field of therapeutic gene editing is rapidly developing with the emergence of CRISPR/Cas9 as well as chemically modified mRNA therapeutics. These new types of nucleic acid therapies are also a good fit with delivery by non‐viral delivery approaches which has led to a renewed interest in lipid‐based and other nanoformulations.