Adeno-Associated Virus (AAV) gene therapy for cystic fibrosis: current barriers and recent developments
نویسندگان
چکیده
منابع مشابه
Barriers and recent advances in non-viral vectors targeting the lungs for cystic fibrosis gene therapy
Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in CFTR genes that affect chloride ion channel. The CF is a good nominee for gene therapy as the asymptomatic carriers are phenotypically normal, and the desired cells are accessible for vector delivery. Gene therapy shows promising effects involving the correction of gene or replacement of the mutant gene with the func...
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ژورنال
عنوان ژورنال: Expert Opinion on Biological Therapy
سال: 2017
ISSN: 1471-2598,1744-7682
DOI: 10.1080/14712598.2017.1347630