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 in cystic fibrosis transfers the noraml CFTR gene to the cells. The sucess of Gene therapy depends on the enrolled patients. The pros for cystic fibrosis gene theray are -

The disadvantage is the airways are difficult to treat. The other disadavtage is getting the right spot on the chromosome.

The patient selection critiera is a important for the sucess to gene therapy. The inclusion criteria are-

With the recent new advances in treatment and progress in the research of gene therapy delivery mechanisms lets hope for a cure in the future.

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