Nature Communications (Aug 2020)
A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency
- Martin H. Kang,
- Laura P. van Lieshout,
- Liqun Xu,
- Jakob M. Domm,
- Arul Vadivel,
- Laurent Renesme,
- Christian Mühlfeld,
- Maria Hurskainen,
- Ivana Mižíková,
- Yanlong Pei,
- Jacob P. van Vloten,
- Sylvia P. Thomas,
- Claudia Milazzo,
- Chanèle Cyr-Depauw,
- Jeffrey A. Whitsett,
- Lawrence M. Nogee,
- Sarah K. Wootton,
- Bernard Thébaud
Affiliations
- Martin H. Kang
- Sinclair Center for Regenerative Medicine, Ottawa Hospital Research Institute
- Laura P. van Lieshout
- Department of Pathobiology, Ontario Veterinary College, University of Guelph
- Liqun Xu
- Sinclair Center for Regenerative Medicine, Ottawa Hospital Research Institute
- Jakob M. Domm
- Department of Pathobiology, Ontario Veterinary College, University of Guelph
- Arul Vadivel
- Sinclair Center for Regenerative Medicine, Ottawa Hospital Research Institute
- Laurent Renesme
- Sinclair Center for Regenerative Medicine, Ottawa Hospital Research Institute
- Christian Mühlfeld
- Institute of Functional and Applied Anatomy, Hannover Medical School
- Maria Hurskainen
- Sinclair Center for Regenerative Medicine, Ottawa Hospital Research Institute
- Ivana Mižíková
- Sinclair Center for Regenerative Medicine, Ottawa Hospital Research Institute
- Yanlong Pei
- Department of Pathobiology, Ontario Veterinary College, University of Guelph
- Jacob P. van Vloten
- Department of Pathobiology, Ontario Veterinary College, University of Guelph
- Sylvia P. Thomas
- Department of Pathobiology, Ontario Veterinary College, University of Guelph
- Claudia Milazzo
- Sinclair Center for Regenerative Medicine, Ottawa Hospital Research Institute
- Chanèle Cyr-Depauw
- Sinclair Center for Regenerative Medicine, Ottawa Hospital Research Institute
- Jeffrey A. Whitsett
- Divisions of Neonatology and Pulmonary Biology, Cincinnati Children’s Hospital Medical Center and University of Cincinnati College of Medicine
- Lawrence M. Nogee
- Division of Neonatology, Department of Pediatrics, Johns Hopkins University School of Medicine
- Sarah K. Wootton
- Department of Pathobiology, Ontario Veterinary College, University of Guelph
- Bernard Thébaud
- Sinclair Center for Regenerative Medicine, Ottawa Hospital Research Institute
- DOI
- https://doi.org/10.1038/s41467-020-17577-8
- Journal volume & issue
-
Vol. 11,
no. 1
pp. 1 – 20
Abstract
Surfactant protein B (SP-B) deficiency is a genetic lung disease that results in lethal respiratory distress within months of birth. Here, the authors describe a gene therapy strategy using a rationally designed AAV6 capsid that restores surfactant homeostasis, prevents lung injury, and improves survival in a mouse model of SP-B deficiency.
