Tinsley J M, Davies K E
Molecular Genetics Group, John Radcliffe Hospital, Headington, Oxford, U.K.
Neuromuscul Disord. 1993 Sep-Nov;3(5-6):537-9. doi: 10.1016/0960-8966(93)90111-v.
This paper reviews the evidence that utrophin, the autosomally encoded protein related to dystrophin, may be capable of performing the same cellular functions as dystrophin. If this is the case, it may be possible to modify the regulation of utrophin expression as an alternative route to dystrophin gene therapy for sufferers of DMD and/or BMD.
本文综述了有关证据,即与抗肌萎缩蛋白相关的常染色体编码蛋白—— utrophin,可能具备与抗肌萎缩蛋白相同的细胞功能。如果真是如此,那么对于杜氏肌营养不良症(DMD)和/或贝克型肌营养不良症(BMD)患者而言,有可能通过改变utrophin表达的调控方式,作为抗肌萎缩蛋白基因疗法的替代途径。