Pioneering Therapies Reshaping Healthcare
Revolutionising medicine: The dawn of gene editing
Gene editing, particularly using the CRISPR technology, has recently gained significant attention in the field of medicine. The US has approved the world’s first gene-editing therapy, which has the potential to transform the medical industry by correcting mutations that cause various diseases.
The approved treatment, known as “exa-cell,” targets sickle cell disease, and there are approximately 280 gene-editing therapies in development, with a focus on hereditary diseases like cystic fibrosis and certain cancers.
Gene Editing for Cystic Fibrosis
Cystic fibrosis (CF) is a genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Gene editing, specifically CRISPR genome editing, holds promise as a potential therapy for CF. The CRISPR gene editing tools, including a “guide” that locates the mutated sequence in the CFTR gene, have shown potential in correcting the mutations that cause CF.
However, there are challenges associated with the effectiveness, efficiency, and long-term safety of gene editing for CF.
Gene Therapy for Cystic Fibrosis
In addition to gene editing, gene therapy, which involves the delivery of a correct version of the CFTR gene into a patient’s cells, is also being explored as a potential treatment for CF. There are two types of gene therapy: integrating and non-integrating. Integrating gene therapy, while promising, poses potential risks such as increasing the risk of cancer.
Non-integrating gene therapy, on the other hand, offers the advantage of not integrating the new genetic material into the patient’s DNA, potentially reducing the risk of long-term side effects.
CRISPR/Cas9 Approach for Cystic Fibrosis Treatment
The CRISPR/Cas9 approach has shown promise in addressing the genetic mutations that cause CF by editing a patient’s genetics. Research and development in this area are ongoing, with a focus on developing CRISPR/Cas9-based medicines for the treatment of CF.
The approach involves a novel protein-RNA complex designed to correct the mutations themselves, offering potential for the treatment of CF and other genetic diseases.
Gene editing, particularly using CRISPR technology, and gene therapy hold significant promise for the treatment of genetic diseases such as cystic fibrosis. While there are challenges and potential risks associated with these approaches, ongoing research and development are paving the way for potential transformative treatments in the field of medicine.
https://www.cff.org/research-clinical-trials/gene-editing-cystic-fibrosis[2] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7313467/
