A team from the Broad Institute of MIT and Harvard, along with Harvard University and the Howard Hughes Medical Institute, has made exciting strides in using a gene-editing tool called prime editing to tackle the mutation responsible for cystic fibrosis in human lung cells. This innovative approach offers a more precise and safer alternative to traditional CRISPR methods, significantly lowering the chances of unintended changes to the genome.
Cystic fibrosis is a genetic disorder that severely affects the lungs and other organs, often leading to chronic breathing problems and persistent infections. Current treatments aim to manage symptoms and include inhaled medications that thin mucus, antibiotics for lung infections, chest physiotherapy for airway clearance, and CFTR modulators that help some patients by restoring protein function. However, these treatments are not cures and require daily management.
The beauty of prime editing lies in its potential to correct the underlying genetic issue, which could vastly improve the quality of life for those living with cystic fibrosis. In their experiments, the researchers successfully corrected the most common mutation in the CFTR gene, restoring normal protein function in lung cells. This promising step forward shows how gene therapy could lead to long-lasting solutions.
Created: Sept 11th, 2024
Citations:
DiCorato, A. (2024, July 10). Prime editing efficiently corrects cystic fibrosis mutation in human lung cells. Broad Institute.
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