Related Experiment Video
Updated: Sep 1, 2025
07:04
Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
Published on: February 11, 2017
19.4K
Cystic Fibrosis Modulator Therapies
Shijing Jia1, Jennifer L Taylor-Cousar2
1Division of Pulmonary and Critical Care Medicine, University of Michigan, Ann Arbor, Michigan, USA;
Annual Review of Medicine
|August 16, 2022
Summary
Cystic fibrosis (CF) treatments have evolved from managing organ damage to targeting specific protein defects. New modulator therapies offer improved outcomes for individuals with CF.
Area of Science:
- Medical Genetics
- Pulmonology
- Gastroenterology
Background:
- Cystic fibrosis (CF) is a genetic disorder affecting multiple organ systems.
- Traditional CF care involved managing symptoms and organ damage.
- Limited treatment options existed for the underlying cause of CF.
Purpose of the Study:
- To highlight the paradigm shift in cystic fibrosis treatment.
- To introduce the impact of novel modulator therapies.
- To discuss the transformation in patient prognosis.
Main Methods:
- Review of traditional and modern therapeutic approaches for CF.
- Focus on the mechanism of action of modulator therapies.
- Analysis of the impact on patient outcomes and quality of life.
Main Results:
- Modulator therapies target specific CFTR protein defects.
- These therapies have significantly improved lung function and reduced complications.
- Patient prognosis and quality of life have been dramatically enhanced.
Conclusions:
- Highly effective modulator therapies represent a breakthrough in CF care.
- Targeting the root cause of CF has transformed patient outcomes.
- Future research should continue to refine these therapies for broader application.
Related Concept Videos
01:24Cystic Fibrosis: Management
216
Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
Sinus disease and chronic...
Sinus disease and chronic...
216
01:23Cystic Fibrosis: Pathogenesis
348
Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation,...
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation,...
348
01:19Antiasthma Drugs: Leukotriene Modifiers
429
Leukotriene modifiers, or cysteinyl leukotriene receptor antagonists, are medications used to manage chronic asthma. These agents target specific inflammatory mediators produced during arachidonic acid metabolism, an essential process in generating inflammation in the body.
Leukotriene modifiers work through two distinct mechanisms:
Leukotriene modifiers work through two distinct mechanisms:
429
01:21Drugs for Treatment of Constipation-Predominant IBS
309
Pharmacological therapies for IBS-C are designed to alleviate abdominal discomfort and enhance bowel function. In patients with IBS-C, fiber supplements may help soften stools and decrease straining, but may also lead to increased gas production and bloating. Osmotic laxatives like milk of magnesia are frequently used to soften stools and increase stool frequency in IBS-C patients. In addition, two drugs approved for use in severe IBS-C adult cases are linaclotide (Linzess) and lubiprostone...
309
01:26COPD: Management Using Bronchodilators and Corticosteroids
284
Chronic obstructive pulmonary isease (COPD) involves a group of progressive lung disorders characterized by persistent airflow limitation and chronic respiratory symptoms. Asthma-COPD Overlap Syndrome (ACOS), encompassing features of both asthma and Chronic obstructive pulmonary disease (COPD), is a group of progressive lung disorders that includes chronic bronchitis, emphysema, and refractory (non-reversible) asthma. ACOS leads to complex clinical presentations that combine the inflammatory...
284
01:29Chronic Obstructive Pulmonary Disease-V: Management
2.7K
Managing Chronic Obstructive Pulmonary Disease (COPD) involves a multifaceted approach to reduce symptoms, prevent exacerbations, improve overall health status, and slow disease progression. Key strategies include lifestyle modifications, pharmacotherapy, supportive therapies, and, in some cases, surgery. Here is an overview of the primary COPD management strategies:
Smoking Cessation
Smoking Cessation
2.7K