13941 - One Strategy for Gene/Cell Therapy: Harness the...

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Unformatted text preview: One Strategy for Gene/Cell Therapy: Harness the Natural Ability of Adult Stem Cells to Repair Tissues Darwin J. Prockop, M.D., Ph.D., Director, Center for Gene Therapy, Tulane University Health Sciences Center, New Orleans, LA How does the body repair itself? Cohnheim (1867):---probably by cells that come from the blood stream. More recent hypothesis---probably first by the stem-like cells found in many tissues.---probably after these are exhausted---by stem cells from the bone arrow How well does the repair system work? We are not at the top of the tree of evolution: Hydra, planaria, etc.---complete organism from a few cells. Salamanders and related---complete limbs or tails Mice and rats---difficult to produce permanent damage in most organs. One Strategy for Gene/Cell Therapy Use stem-like cells from bone marrow---to make man more like a rodent or perhaps salamander. Our approach---large numbers of a patients own cells. Bone marrow aspirate---Local anesthesia Skeletal and Related Disease Diseases of CNS Marrow stromal cells---expanded---gene engineered Therapy of Same Patient Questions 1. Are we certain that marrow contains stem cells for non-hematopoietic tissues? 2. If so, which cells? 3. If so, how do the cells repair tissues? Questions 1. Are we certain marrow contains stem cells for non-hematopoietic tissues ? Conclusive date now from a. differentiation of cells in culture. b. trials in animals. b. patients with bone marrow and organ transplants. 2. Which Cells from Marrow? 1. Hematopoietic stem cells (CD 34* or CD 133*)? 2. Side population cells ? 3. Multipotential adult progenitor cells ? 4 . Mesenchymal stem/marrow stromal cells(MSCs)? OR some still unidentified cell? One Candidate for Reparative Cell from Bone Marrow Isolated by ---plating bone marrow on plastic. Most common names:--- marrow stromal cells ---used as feeder layers for HSCs.--- mesenchymal stem cells ---differentiate into most cell types. COMPROMISE: MSCs. Friedenstein, 1976 Adherent cells (MSCs):---1:10,000 to 100,000 of nucleated cells....
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This note was uploaded on 02/23/2012 for the course PHARM 290 taught by Professor Staff during the Fall '10 term at Rutgers.

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13941 - One Strategy for Gene/Cell Therapy: Harness the...

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