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...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	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
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2016
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/626fe0c8c2014a56953805d0567d624d
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:626fe0c8c2014a56953805d0567d624d
record_format	dspace
spelling	oai:doaj.org-article:626fe0c8c2014a56953805d0567d624d2021-12-02T15:34:58ZIn vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery10.1038/ncomms133042041-1723https://doaj.org/article/626fe0c8c2014a56953805d0567d624d2016-10-01T00:00:00Zhttps://doi.org/10.1038/ncomms13304https://doaj.org/toc/2041-1723Gene 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.Raman BahalNicole Ali McNeerElias QuijanoYanfeng LiuParker SulkowskiAudrey TurchickYi-Chien LuDinesh C. BhuniaArunava MannaDale L. GreinerMichael A. BrehmChristopher J. ChengFrancesc López-GiráldezAdele RicciardiJagadish BeloorDiane S. KrausePriti KumarPatrick G. GallagherDemetrios T. BraddockW. Mark SaltzmanDanith H. LyPeter M. GlazerNature PortfolioarticleScienceQENNature Communications, Vol 7, Iss 1, Pp 1-14 (2016)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q 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
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.
format	article
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
title	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_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_sort	in vivo correction of anaemia in β-thalassemic mice by γpna-mediated gene editing with nanoparticle delivery
publisher	Nature Portfolio
publishDate	2016
url	https://doaj.org/article/626fe0c8c2014a56953805d0567d624d
work_keys_str_mv	AT ramanbahal invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT nicolealimcneer invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT eliasquijano invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT yanfengliu invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT parkersulkowski invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT audreyturchick invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT yichienlu invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT dineshcbhunia invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT arunavamanna invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT dalelgreiner invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT michaelabrehm invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT christopherjcheng invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT francesclopezgiraldez invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT adelericciardi invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT jagadishbeloor invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT dianeskrause invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT pritikumar invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT patrickggallagher invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT demetriostbraddock invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT wmarksaltzman invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT danithhly invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery AT petermglazer invivocorrectionofanaemiainbthalassemicmicebygpnamediatedgeneeditingwithnanoparticledelivery
_version_	1718386659088662528

In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery

Ejemplares similares