After the discovery of the CFTR gene in 1989, the in vitro proof-of-concept for gene therapy for CF was quickly established in 1990. In 1993, the first of many gene therapy clinical trials attempted to rescue the CF defect in airway epithelia.
How Could gene therapy cure cystic fibrosis?
Gene therapy is a process in which a new, correct version of the CFTR gene would be placed into the cells in a person’s body. Although the mutant copies of the CFTR gene would still be there, the presence of the correct copy would give cells the ability to make normal CFTR proteins.
How has treatment improved for cystic fibrosis patients over the years?
New genetic therapies lead to major advances in treatment of cystic fibrosis. A new therapy for cystic fibrosis (CF) that targets the genetic root of the disease has been shown to dramatically improve patients’ lung function and could benefit the vast majority of patients with the disease.
What was the first disease treated with gene therapy?
Related Stories. The first patient to be treated with gene therapy was a four year old girl treated at the NIH Clinical Center in 1990. She had a congenital disease called adenosine deaminase (ADA) deficiency which severely affects immunity and the ability to fight infections.
Does gene therapy cure all cases of cystic fibrosis?
Gene therapy cannot repair organ damage that has already occurred. Although gene therapy could reduce the symptoms of CF and prevent further damage from occurring, it cannot fix scarring or other permanent damage that happened prior to the treatment.
When was gene therapy introduced?
On September 14, 1990, W. French Anderson and his colleagues at the NIH performed the first approved gene therapy procedure on a four-year-old girl born with severe combined immunodeficiency (SCID) (Anderson, 1990).
