Are they working on a cure for cystic fibrosis?
Current treatments for cystic fibrosis are not suitable for all patients and have a limited effect on this life-threatening disease. But new advances in the field promise to overcome these hurdles. The cause of cystic fibrosis is very straightforward. Its treatment, however, is not.
How is gene product therapy used to treat cystic fibrosis?
Gene-product therapy a success in cystic fibrosis treatment. Gene-product modulating therapy – treatment addressing gene abnormality by acting on the proteins arising from the gene rather than trying to correct the gene itself – has been attracting considerable scientific attention in recent years.
Is there a cure for cystic fibrosis in humans?
Gene editing for CF is currently being tested in cells and animals, and it will be a number of years before it can be safely tested in people. Help us blaze a trail to better treatments and a cure for CF.
Is it possible to use gene therapy for CF?
Gene therapy is now a therapeutic reality for some genetic diseases, but the challenge for the CF field is to convert the extensive preclinical developments into an effective and safe treatment option for people with CF. Although gene therapy has the potential to be efficacious for CF, a range of challenges have been identified.
What kind of mutation does cystic fibrosis have?
People with cystic fibrosis have a mutation in both copies of the cystic fibrosis membrane conductance regulator (CFTR) gene. To correct the CFTR mutations in someone’s DNA, the tools needed for gene editing need to get inside the person’s cells, which is very challenging.
What is the genetic cause of cystic fibrosis?
The cause of cystic fibrosis (CF) is a defect in a gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This gene makes a protein that controls the movement of salt and water in and out of the cells in your body.
What does it mean to be a cystic fibrosis carrier?
A cystic fibrosis carrier is a person who has the mutation that causes cystic fibrosis in his or her genes but does not have the disease. Each person has two cystic fibrosis transmembrane regulator genes. If there is one normal cystic fibrosis transmembrane regulator gene and the other is mutated, the person is a carrier.
What are the most common cystic fibrosis mutations?
CF is caused by a mutation in the gene cystic fibrosis transmembrane conductance regulator (CFTR). The most common mutation, ΔF508, is a deletion (Δ signifying deletion) of three nucleotides that results in a loss of the amino acid phenylalanine (F) at the 508th position on the protein.
What is mRNA therapy?
mRNA therapy is engineered to deliver mRNA encoding natural, functional proteins that replace defective or missing proteins, and has potential advantages, including that it: restores gene expression without entering the cell nucleus or changing the genome;