Abstract
279. Derivation and Functional Analysis of Patient Specific Induced Pluripotent Stem Cells as an In Vitro Model of Chronic Granulomatous Disease
Highlights
Chronic granulomatous disease (CGD) is an inherited disorder of phagocytes in which NADPH oxidase is defective in generating reactive oxygen species
Autologous hematopoietic stem cell (HSC) gene therapy represents an alternative to allogenic bone marrow transplantation (ABMT)
We showed that reprogramming vector-free induced pluripotent stem cells (iPSC) clones continue to express pluripotent markers and are able to form embryoid bodies in vitro and teratomas in immunodeficient mice
Summary
Chronic granulomatous disease (CGD) is an inherited disorder of phagocytes in which NADPH oxidase is defective in generating reactive oxygen species. Autologous hematopoietic stem cell (HSC) gene therapy represents an alternative to ABMT. Over the last few years, researchers were able to generate induced pluripotent stem cells (iPSCs) by reprogramming differentiated cells with retroviral expression of stem cell factors (Oct3/4, Sox2, Klf4 and c-Myc).
Sign-in/Register to view highlights & summary Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
