The current therapies available to treat Cystic fibrosis only treat the symptoms

Question

The current therapies available to treat Cystic fibrosis only treat the symptoms of the disorder. Some research groups are exploring the option of using gene therapy. The process involves inserting a good copy of the gene into the patients' blood stem cells in the bone marrow.

Reflecting on the inheritance pattern of cystic fibrosis (autosomal recessive), gene dosage and sufficiency, please explain whether it might be a good idea for a patient to enroll in a clinical trial for cystic fibrosis gene therapy

Explanation / Answer

Gene therapy will play a key role in the treatment of cystic fibrosis which otherwise remains untreatable and do not have sufficient treatment options. It allows noninvasive access to the lungs. The CF gene, the cystic fibrosis transmembrane conductance regulator (CFTR) plays a key role in the gene therapy for the cystic fibrosis.

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