2 doctors weighed in:

Does gene therapy help a cystic fibrosis sufferer?

2 doctors weighed in
Dr. William Walsh
Addiction Medicine

In brief: Trials

Clinical trials for genetic therapy are underway.
As of this time there is no approved method for gene tx in cf. Kalydeco, (ivacaftor) a new drug, dose reverse the effects of one subtype of cf, but it dose so by aiding the damaged sodium-chloride transporter to get in proper position and function normally.

In brief: Trials

Clinical trials for genetic therapy are underway.
As of this time there is no approved method for gene tx in cf. Kalydeco, (ivacaftor) a new drug, dose reverse the effects of one subtype of cf, but it dose so by aiding the damaged sodium-chloride transporter to get in proper position and function normally.
Dr. William Walsh
Dr. William Walsh
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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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