Human neural stem cell-based gene therapy
in neurological diseases

演 者

Seung U. Kim, MD, PhD
(Department of Neurology, UBC Hospital, University of British Columbia, Vancouver, Canada)

日 時

平成22年4月20日(火)16:00 〜17:00

場 所

基礎研究棟2階 教職員ロビー


Cell replacement therapy and gene transfer to the diseased or injured brain have provided the basis for the development of potentially powerful new therapeutic strategies for a broad spectrum of human neurological diseases. In recent years, neurons and glial cells have successfully been generated from stem cells such as embryonic stem cells, mesenchymal stem cells and neural stem cells, and extensive efforts by investigators to develop stem cell-based brain transplantation therapies have been carried out. Recently we have generated continuously dividing immortalized cell lines of human NSCs by introduction of v-myc oncogenes and these immortalized NSC lines have advantageous characteristics for basic studies on neural development and cell replacement therapy or gene therapy studies. Immortalized human NSCs were genetically manipulated in vitro, survive, integrate into host tissues and differentiate into both neurons and glial cells after transplantation to the intact or damaged animal brain. Both in vivo and in vitro human NSCs were able to differentiate into neurons and glial cells and populate the developing or degenerating CNS. We have previously transplanted human neural stem cells in animal models of neurological diseases including Parkinson disease (PD), Huntington disease (HD), amyotrophic lateral sclerosis (ALS), stroke, spinal cord injury and brain tumors, and produced successful clinical outcome. Steady and solid progress in stem cell research in both basic and pre-clinical settings should support the hope for development of stem cell-based therapies for hard-to-cure neurological diseases.

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