In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle...
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
About this item
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Author / Creator
Bahal, Raman , Ali McNeer, Nicole , Quijano, Elias , Liu, Yanfeng , Sulkowski, Parker , Turchick, Audrey , Lu, Yi-Chien , Bhunia, Dinesh C. , Manna, Arunava , Greiner, Dale L. , Brehm, Michael A. , Cheng, Christopher J. , López-Giráldez, Francesc , Ricciardi, Adele , Beloor, Jagadish , Krause, Diane S. , Kumar, Priti , Gallagher, Patrick G. , Braddock, Demetrios T. , Mark Saltzman, W. , Ly, Danith H. and Glazer, Peter M.
Publisher
London: Nature Publishing Group UK
Journal title
Language
English
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Publication information
Publisher
London: Nature Publishing Group UK
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More information
Scope and Contents
Contents
The blood disorder, β-thalassaemia, is considered an attractive target for gene correction. Site-specific triplex formation has been shown to induce DNA repair and thereby catalyse genome editing. Here we report that triplex-forming peptide nucleic acids (PNAs) substituted at the γ position plus stimulation of the stem cell factor (SCF)/c-Kit pathw...
Alternative Titles
Full title
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
Authors, Artists and Contributors
Author / Creator
Ali McNeer, Nicole
Quijano, Elias
Liu, Yanfeng
Sulkowski, Parker
Turchick, Audrey
Lu, Yi-Chien
Bhunia, Dinesh C.
Manna, Arunava
Greiner, Dale L.
Brehm, Michael A.
Cheng, Christopher J.
López-Giráldez, Francesc
Ricciardi, Adele
Beloor, Jagadish
Krause, Diane S.
Kumar, Priti
Gallagher, Patrick G.
Braddock, Demetrios T.
Mark Saltzman, W.
Ly, Danith H.
Glazer, Peter M.
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_626fe0c8c2014a56953805d0567d624d
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_626fe0c8c2014a56953805d0567d624d
Other Identifiers
ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/ncomms13304
