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Progress in Large Animal Models for Hematopoietic Stem Cell Gene Transfer

学友会セミナー

学友会セミナー

2005年開催 学友会セミナー

開催日時: 平成17年7月4日(月) 16:00~17:00
開催場所: 1号館2階会議室
講  師: Peter Horn, MD, Ph.D.
所  属: ハノーバー医科大学輸血部
演  題: "Progress in Large Animal Models for Hematopoietic Stem Cell Gene Transfer"
概  要:

Genetic modification of hematopoietic stem cells (HSCs) has the potential to cure a wide variety of genetic and malignant diseases affecting the hematopoietic system because HSCs provide a lifelong supply of circulating blood cells of all lineages. Genetic diseases such as Fanconi anemia, hemoglobinopathies, and immunodeficiencies, as well as acquired diseases such as AIDS and cancer could potentially be treated by stem cell gene therapy. Although most of these diseases are curable by allogeneic stem cell transplantation, many patients lack a suitable donor, and the side effects of allogeneic stem cell transplantation such as graft-versus-host-disease make the transplantation of genetically modified autologous cells an attractive alternative. Furthermore, recent studies suggest that gene therapy may also enhance allogeneic stem cell transplantation.

The enormous potential of stem cell directed gene therapy has recently been realized by curing children suffering from X-linked severe combined immunodeficiency(SCID). A new era of medicine appeared to be on the horizon, but enthusiasm was quickly halted after it became evident that this clinical success had not come without severe side effects in form of a leukemia-like syndrome. Thus, more refinement and safety testing will be required before gene therapy can be considered a standard clinical treatment, even for very selected groups of patients.

 

Since stem cell transduction protocols developed in the mouse did not translate into efficient gene transfer protocols in human studies, a number of investigators have turned to large animal models to study stem cell transduction. These studies demonstrated that stem cell transduction efficiencies in large animal models were much more similar to human studies than mouse studies, and suggested that these models might be more appropriate assays for the optimization of clinical gene therapy protocols. Large animal models have since been critical to the clinical success of gene therapy. These models will allow us to address many of the remaining safety concerns and test strategies to overcome them. Many of these studies can only be addressed in large animal models due to the different biology of mouse and human stem cells and the lack of in vitro assays for the characterization of stem cells.

世 話 人: ○分子療法分野 東條 有伸
細胞プロセッシング寄付研究部門 高橋 恒夫