Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease

Published 2018 View Full Article

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Title
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease
Authors
Keywords
gene therapy, sickle cell disease, lentiviral vector, integration, genotoxicity, hematopoietic stem-progenitor cells, stem cell clonality, xenograft, NSG mice
Journal
Molecular Therapy-Methods & Clinical Development
Volume 11, Issue -, Pages 167-179
Publisher
Elsevier BV
Online
2018-11-02
DOI
10.1016/j.omtm.2018.10.014

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