A systems pharmacology model for gene therapy in sickle cell disease

• Main Authors: Bo Zheng (Author), Lucia Wille (Author), Karsten Peppel (Author), David Hagen (Author), Andrew Matteson (Author), Jeffrey Ahlers (Author), James Schaff (Author), Fei Hua (Author), Theresa Yuraszeck (Author), Enoch Cobbina (Author), Joshua F. Apgar (Author), John M. Burke (Author), John Roberts (Author), Raibatak Das (Author)
• Format: Book
• Published: Wiley, 2021-07-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...