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What makes cystic fibrosis a good candidate for gene therapy approaches?
Cystic fibrosis is a single gene disorder viewed as a good candidate for gene therapy because the affected gene is known, the target tissue, the lung, is accessible and less than 50% gene transfer may confer clinical benefit.
Can cystic fibrosis be treated with gene therapy?
The study indicated that the CF gene therapy was safe and resulted in a small improvement in lung function. 1 Additional work is needed to improve this kind of gene therapy so that it will be more effective at treating CF.
What makes something a good candidate for gene therapy?
For you to even consider gene therapy, the answer must be “yes.” For instance, genetic disorders caused by mutations in single genes tend to be good candidates for gene therapy, while diseases involving many genes and environmental factors tend to be poor candidates. Do you know which genes are involved?
Why is TRIKAFTA good?
Trikafta is a CFTR modulator that helps defective CFTR proteins work more effectively. Both elexacaftor and tezacaftor work as correctors. They bind to the faulty CFTR protein and help it fold correctly. This way, cells can shuttle more of it to the membrane instead of degrading it.
What should I not take with TRIKAFTA?
Some medicines can cause unwanted or dangerous effects when used with Trikafta….Your doctor may change your treatment plan if you also use:
- rifabutin or rifampin;
- St. John’s wort; or.
- seizure medicine such as carbamazepine, phenobarbital, or phenytoin.
Is cystic fibrosis (CF) a good candidate for gene therapy?
Cystic fibrosis is a single gene disorder viewed as a good candidate for gene therapy because the affected gene is known, the target tissue, the lung, is accessible and less than 50% gene transfer may confer clinical benefit. The CFTR gene encodes a transmembrane protein that regulates chloride transport, but the pathology,…
When did gene therapy for cystic fibrosis become FDA-approved?
In 1993, the first of many gene therapy clinical trials attempted to rescue the CF defect in airway epithelia. Despite the initial enthusiasm, there is still no FDA-approved gene therapy for CF.
Can cystic fibrosis transmembrane conductance regulator modulators prevent lung disease?
Although this is astounding progress, these cystic fibrosis transmembrane conductance regulator (CFTR) modulators are expensive and require lifetime treatment. A one-time treatment administered early in life for people with CF might prevent the onset of lung disease.
What is the best treatment for cystic fibrosis (CF)?
Therapeutic small molecules [1,2,3] provide benefit to a growing percentage of people with CF. Although this is astounding progress, these cystic fibrosis transmembrane conductance regulator (CFTR) modulators are expensive and require lifetime treatment.