Inside CRISPR’s big leap
Gene editing is no longer science fiction—it’s reshaping medicine in real time. From curing genetic diseases to designing lab-grown organs, CRISPR-Cas9 is at the center of a biomedical revolution.
Q: What is CRISPR-Cas9?
A: CRISPR-Cas9, a gene-editing tool derived from bacterial immune systems, has transformed medicine and research since 2012—powering everything from genome editing in animals to lab-created human organs and disease models.
Q: What’s the first FDA-approved CRISPR treatment?
A: In 2023, the FDA approved Casgevy (exagamglogene autotemcel) to treat sickle cell anemia—the first CRISPR-based medicine and a major gene therapy milestone.
Q: How does Casgevy work, and what are the challenges?
A: Stem cells are edited and reinfused after chemotherapy in a year-long process. High costs and limited access mean only about 40 of 16,000 eligible patients had started treatment by late 2024.
Q: How is CRISPR used in research?
A: Researchers use CRISPR to modify genes in animals and cells to study diseases and test therapies. It’s faster and more affordable than older tools, helping uncover roles of specific genes in processes like viral defense.
Q: What’s next for CRISPR?
A: In vivo gene therapy—editing DNA inside the body—could avoid chemotherapy and improve access. But safety concerns remain, and labs are focused on making CRISPR delivery more precise and effective.