Scientific Advances: Synthetic immunomodulation with a CRISPR super-repressor in vivo

The clinical implementation of gene therapy is complicated by a potential immune response to the vectors used for in vivo delivery. In a recent paper by Moghadan et al. CRISPR-mediated transcriptional repression is exploited to dampen the immune response against AAV in vivo.

Reference: Moghadam, F., LeGraw, R., Velazquez, J.J. et al. (2020) Synthetic immunomodulation with a CRISPR super-repressor in vivo. Nat Cell Biol 22, 1143–1154. https://doi.org/10.1038/s41556-020-0563-3

Keywords: Immunosuppression, Gene therapy, CRISPR, Myd88

Questions? Email: crispr@amsterdamumc.nl