Скачать или смотреть Panel: Ex Vivo Gene Therapy: Using Stem Cells to Treat Genetic Disorders - Florian Eichler, Harvard

PANEL: EX VIVO GENE THERAPY: USING BLOOD STEM CELLS TO TREAT GENETIC DISORDERS

Gene therapy is now a therapeutic reality for some terminal or severely disabling disorders. Ex vivo hematopoietic stem cell gene therapy has the critical advantage to turn the cells into widespread delivery vehicles to obtain stable and sustained expression of a defective protein in all appropriate tissues after a single systemic infusion. This session will highlight the potential of this approach in different disorders illustrating the technologies for hematopoietic stem cell gene-correction and the engagement of stem cells to prevent tissue degeneration.

Hematopoietic Stem Cell Gene Therapy for Cerebral Adrenoleukodystrophy:
Florian Eichler, M.D., Director, Center for Rare Neurological Diseases; Associate Professor, Neurology, Harvard Medical School; Assistant in Neurology, Massachusetts General Hospital