CRISPR-Cas9, a gene-editing technology, allows precise modifications to DNA, offering potential cures for genetic disorders like sickle cell anemia and cystic fibrosis. Recent trials have demonstrated successful correction of mutations in vivo, restoring normal protein function.
Key challenges include off-target effects, where unintended DNA edits occur, and delivery issues, requiring efficient vectors like AAVs. Ethical concerns, such as germline editing, remain debated. Future research focuses on improving specificity and scalability for broader clinical use.
Ashish Vishwakarma, et al. "CRISPR-Based Therapies for Genetic Disorders".
OpenJournal system, VOL 1, Issue 1, Case Study.DOI: https://doi.org/6j6291778342986767