As mentioned in the question, Cystic fibrosis is caused by a mutation in a gene resulting in changes to a protein that regulates the movement of salt in and out of cells.
What is gene therapy:
If we talk about gene therapy, it is a process in which we replace a wrong gene with a right gene.
it is a novel and fascinating area of genetic engineering through which we can treat many genetic disorders including Cystic Fibrosis. However, those experimentation of gene therapy are in trial processes.
So now the question is that why Cystic fibrosis can be treated with gene therapy?
The answer is because we already know that cyctic fibrosis is a genetic disorder that is a genetic defect.
We also know which gene is defected in this case which is called CFTR gene.
We also know the target tissue in which damage is caused due to genetic defect and that tissue is lungs.
So, there are 50% chances that if we transfer correct CFTR gene in place of defected CFTR gene of patient, the disease can be treated.
Current research:
In recent few years, experimentation have been made in which Retroviral vectors have been used to transfer the CFTR gene into cultured cells which means outside the body.
So, if the experiments of same nature are proceeded in future in which CFTR gene is transferred in the body of the patient, it can lead to the division of cells with correct CFTR gene and treatment of disease.
In some cases the CFTR gene has been expressed and the protein has been formed which improved the physical condition of the patient. Some other clinical trials have proved that correct CFTR after transfer into the patient through appropriate vector has succesfully expressed itself.
However, more solid research and clinical trials are required to treat Cystic fibrosis through gene therapy and make it a best and easiest option for the treatment of disease.
