In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
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 leve...
| Main Authors: | , , , , , , , , , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Portfolio
2016-10-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/ncomms13304 |
| _version_ | 1819035690253418496 |
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| author | 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 |
| author_facet | 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 |
| author_sort | Raman Bahal |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-21T07:53:38Z |
| format | Article |
| id | doaj.art-626fe0c8c2014a56953805d0567d624d |
| institution | Directory Open Access Journal |
| issn | 2041-1723 |
| language | English |
| last_indexed | 2024-12-21T07:53:38Z |
| publishDate | 2016-10-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | Nature Communications |
| spelling | doaj.art-626fe0c8c2014a56953805d0567d624d2022-12-21T19:11:01ZengNature PortfolioNature Communications2041-17232016-10-017111410.1038/ncomms13304In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle deliveryRaman Bahal0Nicole Ali McNeer1Elias Quijano2Yanfeng Liu3Parker Sulkowski4Audrey Turchick5Yi-Chien Lu6Dinesh C. Bhunia7Arunava Manna8Dale L. Greiner9Michael A. Brehm10Christopher J. Cheng11Francesc López-Giráldez12Adele Ricciardi13Jagadish Beloor14Diane S. Krause15Priti Kumar16Patrick G. Gallagher17Demetrios T. Braddock18W. Mark Saltzman19Danith H. Ly20Peter M. Glazer21Department of Therapeutic Radiology, Yale UniversityDepartment of Therapeutic Radiology, Yale UniversityDepartment of Biomedical Engineering, Yale UniversityDepartment of Therapeutic Radiology, Yale UniversityDepartment of Therapeutic Radiology, Yale UniversityDepartment of Therapeutic Radiology, Yale UniversityDepartment of Laboratory Medicine, Yale UniversityDepartment of Chemistry and Center for Nucleic Acids Science and Technology (CNAST), Carnegie Mellon UniversityDepartment of Chemistry and Center for Nucleic Acids Science and Technology (CNAST), Carnegie Mellon UniversityProgram in Molecular Medicine, University of Massachusetts Medical SchoolProgram in Molecular Medicine, University of Massachusetts Medical SchoolDepartment of Biomedical Engineering, Yale UniversityYale Center for Genome Analysis (YCGA), Yale UniversityDepartment of Therapeutic Radiology, Yale UniversityDepartment of Internal Medicine, Section of Infectious Disease, Yale UniversityDepartment of Laboratory Medicine, Yale UniversityDepartment of Internal Medicine, Section of Infectious Disease, Yale UniversityDepartment of Pediatrics, Yale UniversityDepartment of Pathology, Yale UniversityDepartment of Biomedical Engineering, Yale UniversityDepartment of Chemistry and Center for Nucleic Acids Science and Technology (CNAST), Carnegie Mellon UniversityDepartment of Therapeutic Radiology, Yale UniversityGene 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.https://doi.org/10.1038/ncomms13304 |
| spellingShingle | 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 In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery Nature Communications |
| title | In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery |
| title_full | In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery |
| title_fullStr | In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery |
| title_full_unstemmed | In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery |
| title_short | In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery |
| title_sort | in vivo correction of anaemia in β thalassemic mice by γpna mediated gene editing with nanoparticle delivery |
| url | https://doi.org/10.1038/ncomms13304 |
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