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Psst! Gene Therapy Research Lives

机译:s!基因疗法研究生活

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In 1990, three men―W. French Anderson, R. Michael Blaese, and Kenneth Culver―led a trial in which the genetically corrected adenosine deaminase (ADA) T cells, belonging to a 4-year-old girl, were returned to her. Today, the 16-year-old teenager is alive and well. It took another decade or so for any accomplishments as dramatic as that first trial to be reported, due in part to a relatively empty toolkit. In April, following a trial of gene therapy that occurred two years prior, French researchers announced that the immune systems of several children severely affected with X-linked severe combined immunodeficiency (SCID) were nearly normal, and that no supplementary therapies were involved. Other, less headline-grabbing reports also occurred, including work on curing fatal congenital diseases, reversing infertility in mice, treating patients with hemophilia, and combining different therapies with gene therapy.
机译:1990年,三名男子W。法国的安德森(Andrew),迈克尔·布莱斯(R. Michael Blaese)和肯尼斯·卡尔弗(Kenneth Culver)主持了一项试验,将经过基因校正的腺苷脱氨酶(ADA)T细胞归还给她,该T细胞属于4岁女孩。如今,这个16岁的少年还活着并且身体健康。由于第一次使用了相对空白的工具包,花了十年左右的时间才取得与第一次试验一样巨大的成就。 4月,在两年前进行的基因治疗试验之后,法国研究人员宣布,几名受到X连锁严重联合免疫缺陷症(SCID)严重影响的儿童的免疫系统几乎是正常的,并且没有涉及任何辅助疗法。其他较少引起关注的报道也包括治疗致命的先天性疾病,逆转小鼠不育症,治疗血友病患者以及将不同疗法与基因疗法相结合的工作。

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