Nature Communications (Jul 2021)
In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
- 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
Affiliations
- Sourav K. Bose
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Brandon M. White
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Meghana V. Kashyap
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Apeksha Dave
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Felix R. De Bie
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Haiying Li
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Kshitiz Singh
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Pallavi Menon
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Tiankun Wang
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Shiva Teerdhala
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Vishal Swaminathan
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Heather A. Hartman
- Center for Fetal Research, Children’s Hospital of Philadelphia
- Sowmya Jayachandran
- Division of Pediatric Cardiology, Children’s Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania
- Prashant Chandrasekaran
- Division of Pediatric Cardiology, Children’s Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania
- Kiran Musunuru
- Cardiovascular Institute, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania
- Rajan Jain
- Department of Medicine, Perelman School of Medicine at the University of Pennsylvania
- David B. Frank
- Division of Pediatric Cardiology, Children’s Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania
- Philip Zoltick
- Center for Fetal Research, Children’s Hospital of Philadelphia
- William H. Peranteau
- Center for Fetal Research, Children’s Hospital of Philadelphia
- DOI
- https://doi.org/10.1038/s41467-021-24443-8
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 16
Abstract
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 mutation.
