3’ UTR-truncated HMGA2 overexpression induces non-malignant in vivo expansion of hematopoietic stem cells in non-human primates

3’ UTR-truncated HMGA2 overexpression induces non-malignant in vivo expansion of hematopoietic stem cells in non-human primates

Vector-mediated mutagenesis remains a major safety concern for many gene therapy clinical protocols. Indeed, lentiviral-based gene therapy treatments of hematologic disease can result in oligoclonal blood reconstitution in the transduced cell graft. Specifically, clonal expansion of hematopoietic st...

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Bibliographic Details
Main Authors: Melissa A. Bonner, Antonio Morales-Hernández, Sheng Zhou, Zhijun Ma, Jose Condori, Yong-Dong Wang, Soghra Fatima, Lance E. Palmer, Laura J. Janke, Stephanie Fowler, Brian P. Sorrentino, Shannon McKinney-Freeman
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene therapy
HMGA2
clonal expansion
hematopoietic stem cells
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000796

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http://www.sciencedirect.com/science/article/pii/S2329050121000796

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