Ayan Banerjee

Can genetic editing, specifically base or prime editing, become the primary, curative treatment for all or some types of Cystic Fibrosis, especially in North America and Northwestern Europe?

Started May 29, 2025

Abstract or project description

Cystic Fibrosis is also an autosomal recessive disease where one copy of a mutated CFTR gene is inherited from each parent causing a restricted flow of salt and water in and out of the patient’s lungs. Additionally, CF has 5 different classes of the disease, each characterized by their respective effect on the CFTR protein and the disease. Classes I, II, and III are generally more severe than Classes IV and V, so it is a lot more practical to discuss rolling out advanced treatments for patients with a Class I or II type mutation. This could be a potential ethical controversy if researchers were developing gene editing techniques for CF only to treat a specific portion of the affected populations. Still, though Cystic Fibrosis is a genetically complex disease, which means it may take longer to get new genetic treatments clinically approved, base and prime editing's advantages allow it target the gene rather than the protein, giving it potential to be a promising, one-time and permanent dosage. Thus, while there are many advantages to trying to make base editing as the primary treatment and prevention for Cystic Fibrosis, there are some obstacles that cannot be easily answered.