Add to ORKGSince the cloning of the cystic fibrosis gene (CFTR) in 1989, 18 clinical trials have been carried out, including five in the 2 years reviewed here. Most trials demonstrated proof-of-principle for gene transfer to the airway. However, gene transfer efficiency with each of the three gene transfer agents (adenovirus (Ad), adeno-associated virus 2 (AAV2
Cystic Fibrosis (CF) is the most common, fatal autosomal recessive disorder affecting the Caucasian ...
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has b...
Cystic fibrosis (CF) is the most common monogenic life-threatening disease in the Caucasian populati...
Gene therapy is being developed as a novel treatment for cystic fibrosis (CF), a condition that has ...
Abstract: Cystic fibrosis is a monogenic disorder with significant morbidity and mortality, despite ...
Cystic fibrosis (CF) was one of the first inherited disorders for which gene therapy was seriously c...
Abstract: Gene therapy is being developed as a novel treatment for cystic fibrosis (CF), a condition...
Abstract The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the...
PURPOSE OF THE REVIEW: Since cloning of the disease-causing gene 27 years ago, the development of cy...
Cystic Fibrosis (CF) is an autosomal recessive disorder due to mutations in the CF transmembrane con...
Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders su...
Gene therapy has not yet met the high expectations of “the early days. ” However, it is a promising ...
The initial concept of human gene therapy was permanent cor-rection of a recessive disease by transp...
Airway disease in cystic fibrosis (CF) is the major cause of death and is presently inadequately tre...
Clinical trials in cystic fibrosis (CF) patients established proof-of-principle for transfer of the ...
Cystic Fibrosis (CF) is the most common, fatal autosomal recessive disorder affecting the Caucasian ...
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has b...
Cystic fibrosis (CF) is the most common monogenic life-threatening disease in the Caucasian populati...
Gene therapy is being developed as a novel treatment for cystic fibrosis (CF), a condition that has ...
Abstract: Cystic fibrosis is a monogenic disorder with significant morbidity and mortality, despite ...
Cystic fibrosis (CF) was one of the first inherited disorders for which gene therapy was seriously c...
Abstract: Gene therapy is being developed as a novel treatment for cystic fibrosis (CF), a condition...
Abstract The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the...
PURPOSE OF THE REVIEW: Since cloning of the disease-causing gene 27 years ago, the development of cy...
Cystic Fibrosis (CF) is an autosomal recessive disorder due to mutations in the CF transmembrane con...
Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders su...
Gene therapy has not yet met the high expectations of “the early days. ” However, it is a promising ...
The initial concept of human gene therapy was permanent cor-rection of a recessive disease by transp...
Airway disease in cystic fibrosis (CF) is the major cause of death and is presently inadequately tre...
Clinical trials in cystic fibrosis (CF) patients established proof-of-principle for transfer of the ...
Cystic Fibrosis (CF) is the most common, fatal autosomal recessive disorder affecting the Caucasian ...
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has b...
Cystic fibrosis (CF) is the most common monogenic life-threatening disease in the Caucasian populati...