CRISPR-Cas9 in vivo gene editing: a new frontier

Since its development, CRSIPR-Cas9 gene editing technology has revolutionized biomedical research and opened new possibilities for efficient and highly targeted gene therapies. Martin Ingelsson, MD, PhD, Uppsala University, Uppsala, Sweden, highlights recent major developments in this space and how they are paving the way for the application of CRSIPR-Cas9 to neurodegenerative diseases in the future. In recent years, CRISPR-Cas9 in vivo gene editing has been trialed in clinical studies for the first time in Leber’s congenital amaurosis 10 and transthyretin amyloidosis. While investigations are ongoing, these trials represent a substantial step forward in the gene therapy space. This interview took place at the Harvard European Alumni Training Network (HEAT-Net) 2021 meeting.