Shah Ruth R, Bird Adrian P
Wellcome Trust Centre for Cell Biology, University of Edinburgh, Max Born Crescent, Edinburgh, EH16 5DS, UK.
Genome Med. 2017 Feb 17;9(1):17. doi: 10.1186/s13073-017-0411-7.
Rett syndrome is a profound neurological disorder caused by mutations in the MECP2 gene, but preclinical research has indicated that it is potentially treatable. Progress towards this goal depends on the development of increasingly relevant model systems and on our improving knowledge of MeCP2 function in the brain.
雷特综合征是一种由MECP2基因突变引起的严重神经障碍，但临床前研究表明它具有潜在的可治疗性。实现这一目标的进展取决于越来越相关的模型系统的开发以及我们对MeCP2在大脑中功能的认识的提高。
