Add to ORKGSince identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has been actively developed. More recently gene therapy has been joined by other forms of "genetic medicines" including mRNA delivery, as well as genome editing and mRNA repair-based strategies. Proof-of-concept that gene therapy can stabilize the progression of CF lung disease has recently been established in a Phase IIb trial. An early phase study to assess the safety and explore efficacy of CFTR mRNA repair is ongoing, while mRNA delivery and genome editing-based strategies are currently at the pre-clinical phase of development. This review has been written jointly by some of those involved in the various CF "genetic medicine" fields and will s...
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the membrane protein c...
Many unique genetic procedures have been created to reach the heart of the cystic fibrosis (CF) prob...
Cystic fibrosis (CF) is a monogenic autosomal recessive disorder caused by mutations in the CFTR gen...
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has b...
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has b...
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has b...
Gene therapy is being developed as a novel treatment for cystic fibrosis (CF), a condition that has ...
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis tr...
Abstract Since the early days of its conceptualization and application, human gene transfer held th...
Abstract The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the...
Introduction: Cystic fibrosis (CF) remains a life-threatening genetic disease, with few clinically e...
Cystic fibrosis (CF) was one of the first inherited disorders for which gene therapy was seriously c...
Cystic Fibrosis (CF) is an autosomal recessive disorder due to mutations in the CF transmembrane con...
Abstract The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for ...
Cystic fibrosis (CF) is a progressive, chronic and debilitating genetic disease caused by mutations ...
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the membrane protein c...
Many unique genetic procedures have been created to reach the heart of the cystic fibrosis (CF) prob...
Cystic fibrosis (CF) is a monogenic autosomal recessive disorder caused by mutations in the CFTR gen...
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has b...
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has b...
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has b...
Gene therapy is being developed as a novel treatment for cystic fibrosis (CF), a condition that has ...
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis tr...
Abstract Since the early days of its conceptualization and application, human gene transfer held th...
Abstract The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the...
Introduction: Cystic fibrosis (CF) remains a life-threatening genetic disease, with few clinically e...
Cystic fibrosis (CF) was one of the first inherited disorders for which gene therapy was seriously c...
Cystic Fibrosis (CF) is an autosomal recessive disorder due to mutations in the CF transmembrane con...
Abstract The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for ...
Cystic fibrosis (CF) is a progressive, chronic and debilitating genetic disease caused by mutations ...
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the membrane protein c...
Many unique genetic procedures have been created to reach the heart of the cystic fibrosis (CF) prob...
Cystic fibrosis (CF) is a monogenic autosomal recessive disorder caused by mutations in the CFTR gen...