Seminar & Symposium/Admissions





講演者:  大森 司(自治医科大学医学部 生化学講座 病態生化学部門 教授)

演題: 遺伝性血栓・出血性疾患に対する遺伝子治療・ゲノム編集治療」


日時: 2023年12月19日(火)15:00- 16:00

場所:奥窪記念ホール (臨床医学教育研究センター 1階)





Recently, gene therapy and genome editing have attracted attention as new drug modalities for the treatment of congenital diseases. An adeno-associated virus (AAV) vector-based gene therapy drug for spinal muscular atrophy has been approved in Japan and is now in clinical use. AAV vector-based drugs for hemophilia have also recently been approved by EMA and FDA. In addition, genome editing treatments are now being tested in clinical trials. In this seminar, I will give a general overview of gene therapy and genome editing therapy and introduce recent data on basic research of gene therapy and genome editing treatment in our laboratory. Currently, the drug targets have largely shifted from small molecule compounds to biologics. Japan’s annual biopharmaceutical trade deficit exceeds 3 trillion yen, and this trend is expected to continue in the future. To revitalize the domestic industry and keep up with the wave of globalization, we need to actively develop treatments targeting rare intractable diseases and create new drugs by cooperating with industry, government and academia.


Reference (2022~2023)

担当分野: 幹細胞誘導 江良(内線:6589

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