In vivo base editing rescues Hutchinson-Progeria in a transgenic mouse model.

In vivo base editing rescues Hutchinson-Progeria in a transgenic mouse model. Koblan et al. used AAV9 delivery of an adenine base editor to correct the dominant negative progerin allele in a transgenic mouse harboring the human disease allele. Enduring correction of the allele was achieved with robust reduction in progerin in multiple clinically relevant tissues, improved aortic health, and near normal median lifespan and vitality in mice treated at a young age.