Date of Completion
Spring 5-1-2024
Thesis Advisor(s)
Dr. Charles Giardina; Dr. Stacey L. Hanlon
Honors Major
Molecular and Cell Biology
Disciplines
Cell and Developmental Biology
Abstract
This literature review explores the utilization of CRISPR-Cas9 gene editing technology in addressing Sickle Cell Disease (SCD) and Beta-Thalassemia. CRISPR, leveraging the Cas-9 enzyme inspired by bacterial defense mechanisms, serves as a precise molecular tool for targeted genetic modifications. The process involves guide RNA directing Cas-9 to specific DNA sites, allowing for meticulous gene editing. Focusing on the specific applications of CRISPR in SCD and Beta-Thalassemia, the review critically analyzes recent methods, including CRISPR-Cas9 editing targeting the BCL11A enhancer for SCD and the HBG1/HBG2 promoters for both SCD and Beta-Thalassemia. The synthesis of findings highlights the potential and challenges of these approaches, providing insights into the transformative impact of CRISPR technology on addressing monogenic disorders. While CRISPR-Cas9 holds promising prospects for revolutionizing the treatment of monogenic disorders like SCD and Beta-Thalassemia, ongoing technological limitations warrant further exploration and refinement.
Recommended Citation
Battini, Swetha, "Advancements in CRISPR-Cas9 Gene Editing: Applications and Future Implications for Sickle Cell Disease and β-Thalassemia Treatment" (2024). Honors Scholar Theses. 999.
https://digitalcommons.lib.uconn.edu/srhonors_theses/999