Avances de la terapia CRISPR CAS9 en Fibrosis Quística

Abstract

Advances in clustered regularly interspaced short palindromic repeats associated with protein 9 therapy have boosted forward research of cystic fibrosis, a weakening genetic disease. This gene-editing technique holds promise for correcting the mutations responsible, offering an exciting prospect for patients. However, this technology is still in clinical trials, as regulatory and safety challenges are being faced, and efforts are ongoing to deal with its limitations by optimizing efficiency, reducing off-target effects, customizing approaches, and developing specific delivery methods. Therefore, continuous research and technique optimization are essential to bring advances from the laboratory to successfully apply them in clinical practice, as in recent years, tests have shown promising results, demonstrating that application in humans could be possible within 5 to 10 years.