Gene transfer for cystic fibrosis
نویسندگان
چکیده
منابع مشابه
Gene transfer for cystic fibrosis.
years ago the cystic fibrosis (CF) gene was cloned. The discovery of the gene encoding the CF transmembrane conductance regulator (CFTR) Cl – channel marked the opening of Chapter 1 in the development of gene transfer as a treatment for this common autosomal recessive disease. One year later, Chapter 1 ended when expression of CFTR in CF epithelial cells corrected the CF Cl – transport defect. ...
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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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متن کاملGene therapy for cystic fibrosis.
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ژورنال
عنوان ژورنال: Journal of Clinical Investigation
سال: 1999
ISSN: 0021-9738
DOI: 10.1172/jci8634