7 doctors weighed in:

How does gene therapy help cystic fibrosis?

7 doctors weighed in
Dr. HARRY OPSIMOS
Pediatrics - Pulmonology
6 doctors agree

In brief: Not available yet

Gene therapy is not available for cystic fibrosis (CF) yet (2012).
Having said that, the underlying concept is that since CF is a genetic disease (meaning there is a defect in a gene), by repairing that defect through gene therapy, one can, hopefully, cure the disease.

In brief: Not available yet

Gene therapy is not available for cystic fibrosis (CF) yet (2012).
Having said that, the underlying concept is that since CF is a genetic disease (meaning there is a defect in a gene), by repairing that defect through gene therapy, one can, hopefully, cure the disease.
Dr. HARRY OPSIMOS
Dr. HARRY OPSIMOS
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David Escobar
Family Medicine

In brief: Not effective yet

As of yet, there are no effective gene therapies for cystic fibrosis.
However, there is a new drug in clinical trials right now that might do just this. The gene therapy works, in general, by "fixing" the underlying genetic defect that causes cystic fibrosis. The cftr protein is at the core of this disease etiology, and gene therapy helps the cftr protein form and operate normally.

In brief: Not effective yet

As of yet, there are no effective gene therapies for cystic fibrosis.
However, there is a new drug in clinical trials right now that might do just this. The gene therapy works, in general, by "fixing" the underlying genetic defect that causes cystic fibrosis. The cftr protein is at the core of this disease etiology, and gene therapy helps the cftr protein form and operate normally.
David Escobar
David Escobar
Answer assisted by David Escobar, Medical Student
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