Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester , Manchester, United Kingdom .
Hum Gene Ther. 2018 Oct;29(10):1098-1105. doi: 10.1089/hum.2018.151.
In the last few years, significant advances have occurred in the preclinical and clinical work toward gene and cell therapy for muscular dystrophy. At the time of this writing, several trials are ongoing and more are expected to start. It is thus a time of expectation, even though many hurdles remain and it is unclear whether they will be overcome with current strategies or if further improvements will be necessary.
在过去的几年中，针对肌肉萎缩症的基因和细胞治疗的临床前和临床工作取得了重大进展。在撰写本文时，正在进行几项试验，预计还会有更多的试验开始。因此，这是一个充满期待的时刻，尽管仍然存在许多障碍，而且不清楚这些障碍是否可以通过当前的策略来克服，或者是否需要进一步改进。
