Sidra Jabeenabout 4 years ago

How a genetic 'CopyCatcher' could open the door to CRISPR-based precision gene therapy

Gene therapies such as Novartis’ spinal muscular atrophy treatment Zolgensma deliver a surrogate copy of a gene to replace a dysfunctional one in the body. The CRISPR gene editing tool could offer an alternative strategy—one that could allow for a mutated gene to be fixed on site.