Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins

A platform for systemic therapeutic transgene expression independent of patient mutations needs a safe and highly transcribed locus. Here the authors ex vivo edit HPSCs using CRISPR-Cas9 to integrate transgenes under the α-globin promoter to achieve erythroid specific expression.

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Autores principales:	Giulia Pavani, Marine Laurent, Anna Fabiano, Erika Cantelli, Aboud Sakkal, Guillaume Corre, Peter J. Lenting, Jean-Paul Concordet, Magali Toueille, Annarita Miccio, Mario Amendola
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2020
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/bd6f18164a234100805135163a84adaa
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spelling	oai:doaj.org-article:bd6f18164a234100805135163a84adaa2021-12-02T16:30:12ZEx vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins10.1038/s41467-020-17552-32041-1723https://doaj.org/article/bd6f18164a234100805135163a84adaa2020-07-01T00:00:00Zhttps://doi.org/10.1038/s41467-020-17552-3https://doaj.org/toc/2041-1723A platform for systemic therapeutic transgene expression independent of patient mutations needs a safe and highly transcribed locus. Here the authors ex vivo edit HPSCs using CRISPR-Cas9 to integrate transgenes under the α-globin promoter to achieve erythroid specific expression.Giulia PavaniMarine LaurentAnna FabianoErika CantelliAboud SakkalGuillaume CorrePeter J. LentingJean-Paul ConcordetMagali ToueilleAnnarita MiccioMario AmendolaNature PortfolioarticleScienceQENNature Communications, Vol 11, Iss 1, Pp 1-13 (2020)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Giulia Pavani Marine Laurent Anna Fabiano Erika Cantelli Aboud Sakkal Guillaume Corre Peter J. Lenting Jean-Paul Concordet Magali Toueille Annarita Miccio Mario Amendola Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
description	A platform for systemic therapeutic transgene expression independent of patient mutations needs a safe and highly transcribed locus. Here the authors ex vivo edit HPSCs using CRISPR-Cas9 to integrate transgenes under the α-globin promoter to achieve erythroid specific expression.
format	article
author	Giulia Pavani Marine Laurent Anna Fabiano Erika Cantelli Aboud Sakkal Guillaume Corre Peter J. Lenting Jean-Paul Concordet Magali Toueille Annarita Miccio Mario Amendola
author_facet	Giulia Pavani Marine Laurent Anna Fabiano Erika Cantelli Aboud Sakkal Guillaume Corre Peter J. Lenting Jean-Paul Concordet Magali Toueille Annarita Miccio Mario Amendola
author_sort	Giulia Pavani
title	Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
title_short	Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
title_full	Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
title_fullStr	Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
title_full_unstemmed	Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
title_sort	ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
publisher	Nature Portfolio
publishDate	2020
url	https://doaj.org/article/bd6f18164a234100805135163a84adaa
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Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins

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