The CRISPR Era and Beyond: Technical Milestones and Future Directions in Sickle Cell Disease Therapeutics
Sickle cell disease (SCD) is a major hereditary blood disorder affecting approximately 8 million people worldwide, historically managed through symptomatic care and small-molecule disease-modifying agents. The pivotal 2023 FDA approvals of lovotibeglogene autotemcel (Lyfgenia) and exagamglogene autotemcel (Casgevy) mar...