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 (CF) is also an autosomal recessive disease where one copy of a mutated Cystic Fibrosis Transmembrane Regulator (CFTR) gene is inherited from each parent and causes a restricted flow of salt and water in and out of the patient’s lungs. 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 and are therefore why mutations of only the first two classes are experimented with in this paper. Current CF treatment can temporarily restore CFTR protein function to some mutations, however, its limited longevity and inability to treat all mutations necessitates the introduction of a permanent CF cure. Thus, this paper used derivatives of CRISPR-Cas9 editing–specifically base and prime editing–to correct the Class II F508del mutation; many other Class II mutations were tested with prime editing as well as nonsense Class I mutations tested with base editing. Additionally, strategic optimization of each editing system was also compared with its most primitive version to observe what is the most effective. Still, while there was scientific promise with both types of editing to become an effective cure, questions surrounding biotech funding, ethical controversies, and gene damage remain unaddressed and unanswered, making it impossible, as of now, to declare it a primary clinical CF treatment.