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LW_BCH4122_Lecture5_2009Sep-1 edit

LW_BCH4122_Lecture5_2009Sep-1 edit - Embryonic...

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(Source of image: http://www.paterson.man.ac.uk/images/groups/scbio1.jpg) (Aorta-Gonado- Mesonephros) Embryonic Hematopoiesis Mouse
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1. HSC Transplantation to treat or even cure some diseases (challenge – insufficient matched donors) - Ex Vivo Expansion: HSCs could be expanded in an appropriate niche and growth factors, and/or under special gene regulation. Current studies are evaluating how cultured HSCs compare to non-cultured stem cells, and how cultured cells engraft. 2. Gene Therapy: Correction of inherited ( e.g. SCID- ADA, sickle cell) or acquired genetic defects of hematopoiesis by transplantation of gene-corrected autologous (self) HSCs. Application of HSC
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3. Treat severe autoimmune disease with autologous HSC transplant. From 1996 to 2007, approximately 1,000 patients have been treated. The majority of autoimmune diseases were multiple sclerosis, systemic sclerosis, systemic lupus erythematosus, rheumatoid arthritis, juvenile idiopathic arthritis, and immune cytopenias. Many patients have experienced long-term disease-free remissions. “Resetting" of autoimmunity is
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