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What is gene therapy for cystic fibrosis (CF)?

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Cystic fibrosis is caused by a defect on a gene known as CFTR. New medicines called “CFTR modulators” can fix this gene so it works like it should.

This treatment isn’t for everyone. Many different gene mutations (changes) cause CF. These medications can only fix a few specific mutations. If your CF is caused by another defect, these medications won’t help.

SOURCES:

Mayo Clinic: “Cystic Fibrosis -- Treatment,” “Cystic Fibrosis -- Symptoms and Causes.”

Cystic Fibrosis Foundation: “Life with CF: Treatments and Therapies,” “Nebulizer Care at Home,” “CFTR Modulator Therapies,” “Chest Physical Therapy,” “Why Participate in a Clinical Trial?” “Weighing the Benefits and Risks,” “Fitness.”

National Heart, Lung, and Blood Institute: “How is Cystic Fibrosis Treated?” “What Are Clinical Trials?”

Johns Hopkins Cystic Fibrosis Center: “Treatments: Pancreas/Gastrointestinal Tract.”

Cold Spring Harbor Perspectives in Medicine: “The Cystic Fibrosis Intestine.”

NHS: “Treating Cystic Fibrosis.”

University of Michigan Medicine: “Bronchodilators for Cystic Fibrosis.”

Cystic Fibrosis Center at Stanford: “Sinusitis.”

Reviewed by Dan Brennan on August 17, 2018

SOURCES:

Mayo Clinic: “Cystic Fibrosis -- Treatment,” “Cystic Fibrosis -- Symptoms and Causes.”

Cystic Fibrosis Foundation: “Life with CF: Treatments and Therapies,” “Nebulizer Care at Home,” “CFTR Modulator Therapies,” “Chest Physical Therapy,” “Why Participate in a Clinical Trial?” “Weighing the Benefits and Risks,” “Fitness.”

National Heart, Lung, and Blood Institute: “How is Cystic Fibrosis Treated?” “What Are Clinical Trials?”

Johns Hopkins Cystic Fibrosis Center: “Treatments: Pancreas/Gastrointestinal Tract.”

Cold Spring Harbor Perspectives in Medicine: “The Cystic Fibrosis Intestine.”

NHS: “Treating Cystic Fibrosis.”

University of Michigan Medicine: “Bronchodilators for Cystic Fibrosis.”

Cystic Fibrosis Center at Stanford: “Sinusitis.”

Reviewed by Dan Brennan on August 17, 2018

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What are clinical trials for cystic fibrosis (CF)?

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