Add to ORKGCystic Fibrosis (CF) is an inherited monogenic autosomal recessive genetic disease that is most commonly seen in the Caucasian population. CF is caused by mutations along the Cystic Fibrosis Transmembrane Regulator (CFTR) gene which is a channel protein that regulates chloride- secretion. Conventional treatments such as physical therapy, antibiotics, and nutritional care only alleviates symptoms but does not provide a cure. New emerging channel potentiators and correctors slowed disease progression for 90% of patients. Gene therapy is an attractive strategy for treating CF as it treats all mutation classes along the CFTR gene. Targeting the airway stem cells for permanent integration of CFTR gene may provide a long-term cure for CF. In thi...
Cystic fibrosis (CF) is an autosomal recessive disease caused by defects in the CF transmembrane con...
Cystic fibrosis (CF) is the most common life-shortening autosomal genetic disorder in Caucasians, af...
Cystic fibrosis (CF) is a genetic disease caused by mutations in the CF transmembrane conductance re...
Cystic fibrosis (CF) is an inherited monogenic disorder, amenable to gene-based therapies. Because C...
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis tr...
Cystic Fibrosis (CF) is one of the most common autosomal recessive genetic diseases. It is caused by...
One of the most revolutionary technologies in recent years in the field of molecular biology is CRIS...
Approximately 2,000 different mutations in CFTR gene have been identified to cause cystic fibrosis (...
Cystic fibrosis (CF) is the most common cause of chronic obstructive lung disease in children and yo...
Cystic fibrosis (CF) is a lethal autosomal recessive disease caused by mutations in the cystic fibro...
Abstract Since CRISPR/Cas9 was harnessed to edit DNA, the field of gene therapy has wi...
The CRISPR-Cas9 system is attractive for gene therapy, as it allows for permanent genetic correction...
Abstract Since the early days of its conceptualization and application, human gene transfer held th...
Cystic fibrosis (CF) is a monogenic autosomal recessive disorder caused by mutations in the CFTR gen...
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis tr...
Cystic fibrosis (CF) is an autosomal recessive disease caused by defects in the CF transmembrane con...
Cystic fibrosis (CF) is the most common life-shortening autosomal genetic disorder in Caucasians, af...
Cystic fibrosis (CF) is a genetic disease caused by mutations in the CF transmembrane conductance re...
Cystic fibrosis (CF) is an inherited monogenic disorder, amenable to gene-based therapies. Because C...
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis tr...
Cystic Fibrosis (CF) is one of the most common autosomal recessive genetic diseases. It is caused by...
One of the most revolutionary technologies in recent years in the field of molecular biology is CRIS...
Approximately 2,000 different mutations in CFTR gene have been identified to cause cystic fibrosis (...
Cystic fibrosis (CF) is the most common cause of chronic obstructive lung disease in children and yo...
Cystic fibrosis (CF) is a lethal autosomal recessive disease caused by mutations in the cystic fibro...
Abstract Since CRISPR/Cas9 was harnessed to edit DNA, the field of gene therapy has wi...
The CRISPR-Cas9 system is attractive for gene therapy, as it allows for permanent genetic correction...
Abstract Since the early days of its conceptualization and application, human gene transfer held th...
Cystic fibrosis (CF) is a monogenic autosomal recessive disorder caused by mutations in the CFTR gen...
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis tr...
Cystic fibrosis (CF) is an autosomal recessive disease caused by defects in the CF transmembrane con...
Cystic fibrosis (CF) is the most common life-shortening autosomal genetic disorder in Caucasians, af...
Cystic fibrosis (CF) is a genetic disease caused by mutations in the CF transmembrane conductance re...