Barlow C, Hirotsune S, Paylor R, Liyanage M, Eckhaus M, Collins F, Shiloh Y, Crawley J N, Ried T, Tagle D, Wynshaw-Boris A
Laboratory of Genetic Disease Research, National Center for Human Genome Research, National Institutes of Health, Bethesda, Maryland 20892, USA.
Cell. 1996 Jul 12;86(1):159-71. doi: 10.1016/s0092-8674(00)80086-0.
A murine model of ataxia telangiectasia was created by disrupting the Atm locus via gene targeting. Mice homozygous for the disrupted Atm allele displayed growth retardation, neurologic dysfunction, male and female infertility secondary to the absence of mature gametes, defects in T lymphocyte maturation, and extreme sensitivity to gamma-irradiation. The majority of animals developed malignant thymic lymphomas between 2 and 4 months of age. Several chromosomal anomalies were detected in one of these tumors. Fibroblasts from these mice grew slowly and exhibited abnormal radiation-induced G1 checkpoint function. Atm-disrupted mice recapitulate the ataxia telangiectasia phenotype in humans, providing a mammalian model in which to study the pathophysiology of this pleiotropic disorder.
通过基因靶向破坏Atm基因座,创建了共济失调毛细血管扩张症的小鼠模型。Atm等位基因被破坏的纯合小鼠表现出生长迟缓、神经功能障碍、由于缺乏成熟配子导致的雄性和雌性不育、T淋巴细胞成熟缺陷以及对γ射线极度敏感。大多数动物在2至4个月大时发生恶性胸腺淋巴瘤。在其中一个肿瘤中检测到几种染色体异常。这些小鼠的成纤维细胞生长缓慢,并表现出异常的辐射诱导G1期检查点功能。Atm基因被破坏的小鼠重现了人类共济失调毛细血管扩张症的表型,为研究这种多效性疾病的病理生理学提供了一个哺乳动物模型。