Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease

Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease

The safety and efficacy of lentivirus-mediated gene therapy was recently demonstrated in five male patients with Fabry disease—a rare X-linked lysosomal storage disorder caused by GLA gene mutations that result in multiple end-organ complications. To evaluate the risks of clonal dominance and leukem...

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Bibliographic Details
Main Authors: Amr H. Saleh, Michael Rothe, Dwayne L. Barber, William M. McKillop, Graeme Fraser, Chantal F. Morel, Axel Schambach, Christiane Auray-Blais, Michael L. West, Aneal Khan, Daniel H. Fowler, C. Anthony Rupar, Ronan Foley, Jeffrey A. Medin, Armand Keating
Format: Article
Language:English
Published: Elsevier 2023-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Fabry disease
clinical trial
gene therapy
lentiviral integration
lentiviral safety
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123000050

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http://www.sciencedirect.com/science/article/pii/S2329050123000050

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