Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives

• 主要な著者: Frank J. T. Staal (著者), Alessandro Aiuti (著者), Marina Cavazzana (著者)
• フォーマット: 図書
• 出版事項: Frontiers Media S.A., 2019-10-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: Connect to this object online.

Gene therapy using patient's own stem cells is rapidly becoming an alternative to allogeneic stem cell transplantation, especially when suitably compatible donors cannot be found. The advent of efficient virus-based methods for delivering therapeutic genes has enabled the development of genetic...