Gene therapy: Myth or reality?
Gene therapy: Myth or reality?
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DOI：
10.1016/j.crvi.2016.04.011
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发表时间：
2016-07-01
影响因子：
2
通讯作者：
Fischer, Alain
中科院分区：
文献类型：
--
作者：
Fischer, Alain
基因疗法已经成为现实，尽管仍然很脆弱。在过去 17 年里，在有限数量的医学病症中取得了临床益处，而病理生理学研究确定这些病症是有利的。它们包括遗传性免疫系统疾病、白细胞营养不良、可能的血红蛋白病、B 型血友病和视网膜营养不良。 B细胞白血病和淋巴瘤的治疗也取得了进展。载体开发的进步和基因编辑的可能使用可能会在未来几年带来重大进展。 (C) 2016 年由 Elsevier Masson SAS 代表科学院出版。
Gene therapy has become a reality, although still a fragile one. Clinical benefit has been achieved over the last 17 years in a limited number of medical conditions for which pathophysiological studies determined that they were favorable settings. They include inherited disorders of the immune system, leukodystrophies, possibly hemoglobinopathies, hemophilia B, and retinal dystrophies. Advances in the treatment of B-cell leukemias and lymphomas have also been achieved. Advances in vector development and possible usage of gene editing may lead to significant advances over the next years. (C) 2016 Published by Elsevier Masson SAS on behalf of Academie des sciences.