Parv Sharma

Comprehensive efficacies of mRNA, CRISPR-Cas9, and gene therapy as Cystic Fibrosis treatments

Started Mar. 6, 2023

Abstract or project description

Cystic fibrosis (CF) is a life-threatening genetic disorder characterized by the production of thick, sticky mucus that obstructs the lungs and pancreatic ducts, leading to severe respiratory and digestive complications. Progress in CF therapies has focused on innovative approaches such as mRNA-based therapies, gene therapy, and gene modification with CRISPR-Cas9. mRNA-based therapies have been promising, as these CF treatments use mRNA to restore the function of the defective cystic fibrosis transmembrane conductance regulator (CFTR) protein. Modified mRNA is delivered into cells to produce functional CFTR protein, improving chloride ion transport and reducing mucus viscosity. Gene therapy is another strategy for CF treatment, involving the delivery of CFTR genes to correct the genetic mutation responsible for CF. The primary method utilizes delivering adenovirus to deliver the therapeutic gene into target cells. The emergence of CRISPR-Cas9 genome editing has revolutionized CF therapy by enabling modifications to the CFTR gene, addressing specific CF-causing mutations. Utilizing CRISPR-Cas9, researchers want to correct mutations, restore CFTR protein function, and potentially provide a permanent cure for CF. Significant progress has been made in developing therapies for CF, including mRNA-based therapies, gene therapy, and gene modification with CRISPR-Cas9. These approaches offer potential benefits in alleviating symptoms, enhancing lung function, and even providing a cure for CF, and to properly understand the alleviations plausible, we must consolidate research from research papers and clinical trials - reviewing efficacies of different treatments to determine the potential usefulness of different treatments.