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

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

Lysosomal storage diseases like mucopolysaccharidosis type I (MPS 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 m...

Full description

Bibliographic Details
Main Authors: 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
Language:English
Published: Nature Publishing Group 2021-07-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-021-24443-8

Internet

https://doi.org/10.1038/s41467-021-24443-8

Similar Items