Junk DNA in birds may hold key to safe, efficient gene therapy
Published Date: 2/20/2024
Source: phys.org
The recent approval of a CRISPR-Cas9 therapy for sickle cell disease demonstrates that gene editing tools can do a superb job of knocking out genes to cure hereditary disease. But it's still not possible to insert whole genes into the human genome to substitute for defective or deleterious genes.