A member asked:

How should gene therapy treat these two diseases. cystic fibrosis and parkinson disease?

A doctor has provided 1 answer
Dr. Lokesh Guglani answered

Specializes in Pediatric Pulmonology

Correct gene defects: Cystic fibrosis is a genetic disorder where a dysfunctional cftr protein causes problems in various organ systems. By restoring the function of this protein in the involved body systems, it is hoped that normal functionality will be restored & the harmful effects of CF disease will improve. There are still several obstacles before it can be successfully implemented in CF patients everywhere.

Answered 6/10/2014

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