Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need
The PAH c.1222C>T variant is the most frequent variant reported in phenylketonuria (PKU) individuals. Here, the authors show these individuals have poor metabolic control, and they use prime editing, delivered by adeno-associated viral vectors, to directly correct the variant in the liver in humanized PKU mice and definitively treat the disease.
Source: The American Journal of Human Genetics - Category: Genetics & Stem Cells Authors: Dominique L. Brooks, Madelynn N. Whittaker, Ping Qu, Kiran Musunuru, Rebecca C. Ahrens-Nicklas, Xiao Wang Tags: Article Source Type: research