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Currently, there is no cure for cystic fibrosis. But some scientists at Yale could change that.
On April 27th, they published a paper explaining how they successfully fixed the most common mutation in the gene that causes cystic fibrosis.
A team led by Dr. Marie Egan, Dr. Peter Glazer, and Mark Saltzman created synthetic molecules similar to DNA called peptide nucleic acids (PNA). They delivered the PNA, along with some donor DNA, into human and mouse airway cells by using nanoparticles billionths of a meter wide. “What the PNA does is clamp to the DNA close to the mutation, triggering DNA repair and recombination pathways in cells,” said Dr. Egan.
This process is known as gene editing. The scientists successfully edited a high percentage of the targeted cells. Not only that, but there were very few unintended effects on the cells.
This could be big news for the more than 70,000 people with cystic fibrosis. Cystic fibrosis is a life threatening genetic disease that affects one’s lungs and digestive system. It can be treated through airway clearance, inhaling medicine through a nebulizer, or taking pancreatic enzyme supplements. However, these treatments are merely symptom management.
According to Dr. Egan, the successful editing of the cystic fibrosis gene is just “step one in a long process.” But it shows promise. “The technology could be used as a way to fix the basic genetic defect in cystic fibrosis.”
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