Brouwer Judith R, Willemsen Rob, Oostra Ben A
Department of Clinical Genetics, ErasmusMC, Rotterdam, The Netherlands.
Bioessays. 2009 Jan;31(1):71-83. doi: 10.1002/bies.080122.
Over 20 unstable microsatellite repeats have been identified as the cause of neurological disease in humans. The repeat nucleotide sequences, their location within the genes, the ranges of normal and disease-causing repeat length and the clinical outcomes differ. Unstable repeats can be located in the coding or the non-coding region of a gene. Different pathogenic mechanisms that are hypothesised to underlie the diseases are discussed. Evidence is given both from studies in simple model systems and from studies on human material and in animal models. Since somatic instability might affect the clinical outcome, this is briefly touched on. Available data and theories on the timing and mechanisms of the repeat instability itself are discussed, along with factors that have been observed to affect instability. Finally, the question of why the often harmful unstable repeats have been maintained throughout evolution is addressed.
20多种不稳定微卫星重复序列已被确定为人类神经疾病的病因。重复核苷酸序列、它们在基因中的位置、正常和致病重复长度范围以及临床结果各不相同。不稳定重复序列可位于基因的编码区或非编码区。文中讨论了假定为这些疾病潜在病因的不同致病机制。既有来自简单模型系统研究的证据,也有来自对人体材料和动物模型研究的证据。由于体细胞不稳定性可能影响临床结果,因此对此也作了简要探讨。文中讨论了关于重复序列不稳定性本身的时间和机制的现有数据及理论,以及已观察到的影响不稳定性的因素。最后,探讨了为何这些往往有害的不稳定重复序列在整个进化过程中得以保留的问题。