In utero nanoparticle delivery for site-specific genome editing

The correction of genetic defects in utero could allow for improved outcomes of gene therapy. Here, the authors demonstrate safe delivery of nanoparticles to fetal mouse tissues, and show that nanoparticles containing peptide nucleic acids to edit the beta-globin gene are effective in a mouse model...

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Autores principales:	Adele S. Ricciardi, Raman Bahal, James S. Farrelly, Elias Quijano, Anthony H. Bianchi, Valerie L. Luks, Rachael Putman, Francesc López-Giráldez, Süleyman Coşkun, Eric Song, Yanfeng Liu, Wei-Che Hsieh, Danith H. Ly, David H. Stitelman, Peter M. Glazer, W. Mark Saltzman
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2018
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/adf52524b8364f4a87460fa79da2c6f9
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spelling	oai:doaj.org-article:adf52524b8364f4a87460fa79da2c6f92021-12-02T17:33:15ZIn utero nanoparticle delivery for site-specific genome editing10.1038/s41467-018-04894-22041-1723https://doaj.org/article/adf52524b8364f4a87460fa79da2c6f92018-06-01T00:00:00Zhttps://doi.org/10.1038/s41467-018-04894-2https://doaj.org/toc/2041-1723The correction of genetic defects in utero could allow for improved outcomes of gene therapy. Here, the authors demonstrate safe delivery of nanoparticles to fetal mouse tissues, and show that nanoparticles containing peptide nucleic acids to edit the beta-globin gene are effective in a mouse model of beta-thalassemia.Adele S. RicciardiRaman BahalJames S. FarrellyElias QuijanoAnthony H. BianchiValerie L. LuksRachael PutmanFrancesc López-GiráldezSüleyman CoşkunEric SongYanfeng LiuWei-Che HsiehDanith H. LyDavid H. StitelmanPeter M. GlazerW. Mark SaltzmanNature PortfolioarticleScienceQENNature Communications, Vol 9, Iss 1, Pp 1-11 (2018)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Adele S. Ricciardi Raman Bahal James S. Farrelly Elias Quijano Anthony H. Bianchi Valerie L. Luks Rachael Putman Francesc López-Giráldez Süleyman Coşkun Eric Song Yanfeng Liu Wei-Che Hsieh Danith H. Ly David H. Stitelman Peter M. Glazer W. Mark Saltzman In utero nanoparticle delivery for site-specific genome editing
description	The correction of genetic defects in utero could allow for improved outcomes of gene therapy. Here, the authors demonstrate safe delivery of nanoparticles to fetal mouse tissues, and show that nanoparticles containing peptide nucleic acids to edit the beta-globin gene are effective in a mouse model of beta-thalassemia.
format	article
author	Adele S. Ricciardi Raman Bahal James S. Farrelly Elias Quijano Anthony H. Bianchi Valerie L. Luks Rachael Putman Francesc López-Giráldez Süleyman Coşkun Eric Song Yanfeng Liu Wei-Che Hsieh Danith H. Ly David H. Stitelman Peter M. Glazer W. Mark Saltzman
author_facet	Adele S. Ricciardi Raman Bahal James S. Farrelly Elias Quijano Anthony H. Bianchi Valerie L. Luks Rachael Putman Francesc López-Giráldez Süleyman Coşkun Eric Song Yanfeng Liu Wei-Che Hsieh Danith H. Ly David H. Stitelman Peter M. Glazer W. Mark Saltzman
author_sort	Adele S. Ricciardi
title	In utero nanoparticle delivery for site-specific genome editing
title_short	In utero nanoparticle delivery for site-specific genome editing
title_full	In utero nanoparticle delivery for site-specific genome editing
title_fullStr	In utero nanoparticle delivery for site-specific genome editing
title_full_unstemmed	In utero nanoparticle delivery for site-specific genome editing
title_sort	in utero nanoparticle delivery for site-specific genome editing
publisher	Nature Portfolio
publishDate	2018
url	https://doaj.org/article/adf52524b8364f4a87460fa79da2c6f9
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In utero nanoparticle delivery for site-specific genome editing

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