Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders

Vectors used in gene therapy for hemoglobin disorders carry globin in a reverse-orientation to prevent the loss of key regulatory elements by RNA splicing, but this limits their efficiency. Here, the authors develop a vector carrying β-globin in a forward orientation and show that it has improved ti...

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Autores principales:	Naoya Uchida, Matthew M. Hsieh, Lydia Raines, Juan J. Haro-Mora, Selami Demirci, Aylin C. Bonifacino, Allen E. Krouse, Mark E. Metzger, Robert E. Donahue, John F. Tisdale
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2019
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/10b9f0823d304489858931b8d82f9d3e
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spelling	oai:doaj.org-article:10b9f0823d304489858931b8d82f9d3e2021-12-02T15:36:18ZDevelopment of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders10.1038/s41467-019-12456-32041-1723https://doaj.org/article/10b9f0823d304489858931b8d82f9d3e2019-10-01T00:00:00Zhttps://doi.org/10.1038/s41467-019-12456-3https://doaj.org/toc/2041-1723Vectors used in gene therapy for hemoglobin disorders carry globin in a reverse-orientation to prevent the loss of key regulatory elements by RNA splicing, but this limits their efficiency. Here, the authors develop a vector carrying β-globin in a forward orientation and show that it has improved titers and transduction efficiency in humanized mice and nonhuman primates.Naoya UchidaMatthew M. HsiehLydia RainesJuan J. Haro-MoraSelami DemirciAylin C. BonifacinoAllen E. KrouseMark E. MetzgerRobert E. DonahueJohn F. TisdaleNature PortfolioarticleScienceQENNature Communications, Vol 10, Iss 1, Pp 1-14 (2019)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Naoya Uchida Matthew M. Hsieh Lydia Raines Juan J. Haro-Mora Selami Demirci Aylin C. Bonifacino Allen E. Krouse Mark E. Metzger Robert E. Donahue John F. Tisdale Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders
description	Vectors used in gene therapy for hemoglobin disorders carry globin in a reverse-orientation to prevent the loss of key regulatory elements by RNA splicing, but this limits their efficiency. Here, the authors develop a vector carrying β-globin in a forward orientation and show that it has improved titers and transduction efficiency in humanized mice and nonhuman primates.
format	article
author	Naoya Uchida Matthew M. Hsieh Lydia Raines Juan J. Haro-Mora Selami Demirci Aylin C. Bonifacino Allen E. Krouse Mark E. Metzger Robert E. Donahue John F. Tisdale
author_facet	Naoya Uchida Matthew M. Hsieh Lydia Raines Juan J. Haro-Mora Selami Demirci Aylin C. Bonifacino Allen E. Krouse Mark E. Metzger Robert E. Donahue John F. Tisdale
author_sort	Naoya Uchida
title	Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders
title_short	Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders
title_full	Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders
title_fullStr	Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders
title_full_unstemmed	Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders
title_sort	development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders
publisher	Nature Portfolio
publishDate	2019
url	https://doaj.org/article/10b9f0823d304489858931b8d82f9d3e
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Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders

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