Nature Communications (Oct 2016)
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
- Raman Bahal,
- Nicole Ali McNeer,
- Elias Quijano,
- Yanfeng Liu,
- Parker Sulkowski,
- Audrey Turchick,
- Yi-Chien Lu,
- Dinesh C. Bhunia,
- Arunava Manna,
- Dale L. Greiner,
- Michael A. Brehm,
- Christopher J. Cheng,
- Francesc López-Giráldez,
- Adele Ricciardi,
- Jagadish Beloor,
- Diane S. Krause,
- Priti Kumar,
- Patrick G. Gallagher,
- Demetrios T. Braddock,
- W. Mark Saltzman,
- Danith H. Ly,
- Peter M. Glazer
Affiliations
- Raman Bahal
- Department of Therapeutic Radiology, Yale University
- Nicole Ali McNeer
- Department of Therapeutic Radiology, Yale University
- Elias Quijano
- Department of Biomedical Engineering, Yale University
- Yanfeng Liu
- Department of Therapeutic Radiology, Yale University
- Parker Sulkowski
- Department of Therapeutic Radiology, Yale University
- Audrey Turchick
- Department of Therapeutic Radiology, Yale University
- Yi-Chien Lu
- Department of Laboratory Medicine, Yale University
- Dinesh C. Bhunia
- Department of Chemistry and Center for Nucleic Acids Science and Technology (CNAST), Carnegie Mellon University
- Arunava Manna
- Department of Chemistry and Center for Nucleic Acids Science and Technology (CNAST), Carnegie Mellon University
- Dale L. Greiner
- Program in Molecular Medicine, University of Massachusetts Medical School
- Michael A. Brehm
- Program in Molecular Medicine, University of Massachusetts Medical School
- Christopher J. Cheng
- Department of Biomedical Engineering, Yale University
- Francesc López-Giráldez
- Yale Center for Genome Analysis (YCGA), Yale University
- Adele Ricciardi
- Department of Therapeutic Radiology, Yale University
- Jagadish Beloor
- Department of Internal Medicine, Section of Infectious Disease, Yale University
- Diane S. Krause
- Department of Laboratory Medicine, Yale University
- Priti Kumar
- Department of Internal Medicine, Section of Infectious Disease, Yale University
- Patrick G. Gallagher
- Department of Pediatrics, Yale University
- Demetrios T. Braddock
- Department of Pathology, Yale University
- W. Mark Saltzman
- Department of Biomedical Engineering, Yale University
- Danith H. Ly
- Department of Chemistry and Center for Nucleic Acids Science and Technology (CNAST), Carnegie Mellon University
- Peter M. Glazer
- Department of Therapeutic Radiology, Yale University
- DOI
- https://doi.org/10.1038/ncomms13304
- Journal volume & issue
-
Vol. 7,
no. 1
pp. 1 – 14
Abstract
Gene editing approaches are widely used for correcting mutations, but their application is largely limited to cells and not living animals. Here the authors show that in vivoγPNA-mediated editing of a β-globin mutation is promoted by SCF and leads to sustained normalization of blood haemoglobin levels β-thalassemic mice.
