Gene Therapy for Cystic Fibrosis
Cystic fibrosis is caused by mutations in the gene responsible for producing the cystic fibrosis transmembrane conductance regulator (CFTR) protein. For this reason, scientists are …
FDA approves new breakthrough therapy for cystic fibrosis
FDA approves breakthrough therapy Trikafta for patients 12 and older with cystic fibrosis who have at least one F508del mutation in the CFTR gene, estimated to represent 90% of the cystic...
Gene therapy for cystic fibrosis: Challenges and prospects
In this review, we look back at the history for the lessons learned from previous clinical trials and summarize the recent advances in the research for CF gene therapy, including the emerging …
The future of cystic fibrosis treatment: from disease mechanisms …
2023年9月30日 · With the 2019 breakthrough in the development of highly effective modulator therapy providing unprecedented clinical benefits for over 90% of patients with cystic fibrosis …
Gene Therapy for Cystic Fibrosis: Lessons Learned and Paths …
For 90% of patients, small-molecule drugs that correct the basic defects in the mutant CFTR channel (i.e., CFTR modulators) are an effective treatment option. However, CF patients …
Can Gene Therapy Help Cystic Fibrosis? - Healthline
2024年5月24日 · Gene therapy for CF involves delivering a correct copy of the cystic fibrosis transmembrane conductance regulator (CFTR) gene into the cells of a person with CF.
Future therapies for cystic fibrosis - Nature Communications
2023年2月8日 · We are currently witnessing transformative change for people with cystic fibrosis with the introduction of small molecule, mutation-specific drugs capable of restoring function of …
Gene therapy for cystic fibrosis: new tools for precision medicine
2021年10月30日 · In this review, we highlight some of the more recent gene therapy approaches as well as new models that will provide insight into personalized therapies for CF. Cystic …
Gene therapy for the treatment of cystic fibrosis - PMC
Gene therapy involves the transfer of correct copies of cystic fibrosis transmembrane conductance regulator (CFTR) DNA to the epithelial cells in the airways. The cloning of the …
Trial of CF gene therapy SP-101 begins dosing, is still recruiting
2024年11月18日 · The therapy delivers its genetic payload using a version of adeno-associated virus, or AAV, that’s been engineered to deliver the therapeutic gene instead of causing …