TALEN-Mediated Gene Targeting for Cystic Fibrosis-Gene Therapy
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...
متن کاملGene therapy for cystic fibrosis.
Following the cloning of the cystic fibrosis (CF) gene, in vitro studies rapidly established the feasibility of gene therapy for this disease. Unlike ex vivo approaches that have been utilized for other genetic diseases such as adenosine deaminase deficiency, gene therapy for CF will likely require direct in vivo delivery of gene transfer vectors to the airways of patients with CF. Hence, major...
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Cystic Fibrosis (CF) is the most common fatal autosomal recessive genetic disease in the Caucasians with a frequency of approximately 1 in 2500 newborns (Cystic Fibrosis Founda‐ tion, http://www.cff.org/). It affects several organs including the lungs, the liver, the pan‐ creas, the sweat glands and the gastrointestinal and reproductive tracts [1]. The most severe complications that finally lea...
متن کاملGene therapy in cystic fibrosis.
Theoretically, cystic fibrosis transmembrane conductance regulator (CFTR) gene replacement during the neonatal period can decrease morbidity and mortality from cystic fibrosis (CF). In vivo gene transfers have been accomplished in CF patients. Choice of vector, mode of delivery to airways, translocation of genetic information, and sufficient expression level of the normalized CFTR gene are issu...
متن کاملToward cystic fibrosis gene therapy.
Cystic fibrosis (CF) is a common genetic disorder characterized by defective epithelial chloride transport and progressive lung disease. Although great strides have been made in the treatment of CF, it remains lethal, often by early adulthood. CF is one of the most extensively researched genetic diseases as a target for gene therapy development. It may also serve as an important model for gene ...
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ژورنال
عنوان ژورنال: Genes
سال: 2019
ISSN: 2073-4425
DOI: 10.3390/genes10010039