In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease

oleh: Sourav K. Bose, Brandon M. White, Meghana V. Kashyap, Apeksha Dave, Felix R. De Bie, Haiying Li, Kshitiz Singh, Pallavi Menon, Tiankun Wang, Shiva Teerdhala, Vishal Swaminathan, Heather A. Hartman, Sowmya Jayachandran, Prashant Chandrasekaran, Kiran Musunuru, Rajan Jain, David B. Frank, Philip Zoltick, William H. Peranteau

Format: Article
Diterbitkan: Nature Portfolio 2021-07-01

Deskripsi

Lysosomal storage diseases like mucopolysaccharidosis type I (MPSĀ I) cause pathology before birth and result in early morbidity and mortality. Here, the authors show that in utero base editing mediates multi-organ phenotypic and survival benefits in a mouse model recapitulating a common human MPSI mutation.