Departments of Ophthalmology and Visual Sciences, University of Massachusetts Medical School, Worcester, Massachusetts, USA.
Departments of Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA.
Hum Gene Ther. 2022 Sep;33(17-18):865-878. doi: 10.1089/hum.2022.159.
In 2001, the first large animal was successfully treated with a gene therapy that restored its vision. Lancelot, the Briard dog that was treated, suffered from a human childhood blindness called Leber's congenital amaurosis type 2. Sixteen years later, the gene therapy was approved by the U.S. Food and Drug Administration. The success of this gene therapy in dogs led to a fast expansion of the ocular gene therapy field. By now every class of inherited retinal dystrophy has been treated in at least one animal model and many clinical trials have been initiated in humans. In this study, we review the status of viral gene therapies for the retina, with a focus on ongoing human clinical trials. It is likely that in the next decade we will see several new viral gene therapies approved.
2001 年，首例大型动物通过基因治疗成功恢复视力。接受治疗的布里犬兰斯洛特患有莱伯先天性黑蒙 2 型，这是一种儿童期失明的人类疾病。16 年后，美国食品和药物管理局批准了这种基因疗法。这种基因疗法在狗身上的成功导致了眼基因治疗领域的快速扩张。到目前为止，每一种遗传性视网膜营养不良都在至少一种动物模型中得到了治疗，并且已经在人类中启动了许多临床试验。在这项研究中，我们回顾了用于视网膜的病毒基因治疗的现状，重点是正在进行的人类临床试验。在未来十年，我们很可能会看到几种新的病毒基因治疗方法获得批准。
