Genetic therapies for cystic fibrosis lung disease


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Abstract

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.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.

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