Related Experiment Video
Updated: Apr 2, 2026
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
20.3K
A combination therapy for cystic fibrosis
Jeffrey L Brodsky1, Raymond A Frizzell2
1Department of Biological Sciences, University of Pittsburgh, Pittsburgh, PA 15260, USA.
Cell
|September 26, 2015
Summary
Cystic fibrosis, a common genetic disorder, is often caused by a specific mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A new treatment combines a corrector and potentiator to improve CFTR protein function.
Area of Science:
- Biochemistry
- Genetics
- Pharmacology
Background:
- Cystic fibrosis is a prevalent genetic disorder.
- The most common form results from an amino acid deletion in the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
Observation:
- A recently approved treatment targets individuals with homozygous mutations for this specific CFTR defect.
Findings:
- The treatment employs a two-component approach: a chemical corrector and a potentiator.
- The corrector facilitates proper CFTR protein folding.
- The potentiator enhances the activity of the CFTR channel.
Implications:
- This therapeutic strategy offers a new avenue for managing cystic fibrosis.
- Improved CFTR function can alleviate symptoms associated with the disease.
More Related Videos
Related Concept Videos
02:50Combination Therapies and Personalized Medicine
6.3K
Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
6.3K
01:27Combined Effects of Drugs: Synergism
7.5K
Synergism is a useful mechanism where combining two or more drugs is more effective than each constituent used alone. Such combinations are also called supra-additive interactions. The drugs collectively enhance the final therapeutic effect by acting on different targets. Another advantage is that the low dose of each constituent drug is sufficient to achieve the desired effect. This helps reduce the duration of therapy and lower the adverse effects of these drugs.
Such synergistic combinations...
Such synergistic combinations...
7.5K
01:23Cystic Fibrosis: Pathogenesis
1.1K
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,...
1.1K
01:24Cystic Fibrosis: Management
620
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...
620
01:21Drugs for Treatment of Constipation-Predominant IBS
1.3K
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...
1.3K
01:28Pulmonary Tuberculosis V
870
Medical management of tuberculosis (TB) patients involves a comprehensive approach that includes diagnosis, treatment, and monitoring. The specific strategies can vary depending on the type of tuberculosis (latent or active), the patient's overall health status, and other considerations.
Latent tuberculosis infection occurs when TB bacteria are present in a person's body, but are not causing illness or symptoms. It is not contagious, and preventive treatment is crucial to avoid the...
Latent tuberculosis infection occurs when TB bacteria are present in a person's body, but are not causing illness or symptoms. It is not contagious, and preventive treatment is crucial to avoid the...
870