A systems pharmacology model for gene therapy in sickle cell disease

Abstract We developed a mathematical model for autologous stem cell therapy to cure sickle cell disease (SCD). Experimental therapies using this approach seek to engraft stem cells containing a curative gene. These stem cells are expected to produce a lifelong supply of red blood cells (RBCs) contai...

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Bibliographic Details
Main Authors: Bo Zheng, Lucia Wille, Karsten Peppel, David Hagen, Andrew Matteson, Jeffrey Ahlers, James Schaff, Fei Hua, Theresa Yuraszeck, Enoch Cobbina, Joshua F. Apgar, John M. Burke, John Roberts, Raibatak Das
Format: Article
Language:English
Published: Wiley 2021-07-01
Series:CPT: Pharmacometrics & Systems Pharmacology
Online Access:https://doi.org/10.1002/psp4.12638