A 46-year-old member asked:
how does gene therapy help cystic fibrosis?
2 doctor answers • 9 doctors weighed in
Dr. HARRY OPSIMOS answered
Pediatric Pulmonology 22 years experience
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.
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Dr. David Escobar answered
Family Medicine 9 years experience
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.
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Similar questions
A 37-year-old member asked:
How can gene therapy help a cystic fibrosis sufferer?
5 doctor answers • 8 doctors weighed in
Dr. HARRY OPSIMOS answered
Pediatric Pulmonology 22 years experience
Not available: There is currently no gene therapy available for cystic fibrosis (cf). Since CF is a genetic disease, there is hope that, one day, gene therapy may provide a cure for this disease by, essentially, correcting the underlying genetic defect.
6256 views
A 29-year-old member asked:
How does gene therapy help a cystic fibrosis patient?
1 doctor answer • 2 doctors weighed in
Dr. Derrick Lonsdale answered
Preventive Medicine 73 years experience
So far not at all: Trying to replace the faulty gene has been a failure so far. The new science of epigenetics tells us that our genes (even "bad" ones) are changed by diet and life style. I havegiven children with CF vitamin supplements. They help greatly but there is no cure. Find a physician who uses nutrient treatment.
5860 views
A 37-year-old member asked:
Theres is no cure for cystic fibrosis so what could gene therapy do?
2 doctor answers • 4 doctors weighed in
Dr. HARRY OPSIMOS answered
Pediatric Pulmonology 22 years experience
Cure: Depending on the type of gene therapy, gene therapy can be a cure, if the underlying genetic problem is corrected. Of course, any damage done to the body by the disease up to that point may, or may not be, reversible.
5842 views
India
A 44-year-old male asked:
Is there gene therapy for cystic fibrosis and health centrefor this disease in the us?
1 doctor answer • 3 doctors weighed in
Dr. Douglas Arenberg answered
Pulmonary Critical Care 31 years experience
No and yes: Safe effective gene therapy for CF is a dream that we have yet to realize, but there are many excellent CF centers in the us. The CF foundation is an excellent source of information on these centers. The treatments for CF are improving, and expanding constantly. The life expectancy for patients with CF has steadily increased but is still limited by pulmonary disease.
5588 views
A 35-year-old member asked:
What is possible cures for cystic fibrosis, how would gene therapy work?
1 doctor answer • 1 doctor weighed in
Dr. Peter Francis answered
Retinal Surgery 29 years experience
Replace gene: In cystic fibrosis the underlying problem is that a single gene is not functioning. Gene therapy would allow that gene to be replaced with a normal functional gene. In theory, this would offer a cure. In practice, such a cure has proven elusive however work continues.
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Last updated Sep 29, 2019
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