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...
Main Authors: | , , , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Wiley
2021-07-01
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Series: | CPT: Pharmacometrics & Systems Pharmacology |
Online Access: | https://doi.org/10.1002/psp4.12638 |