Kirchhausen T
Center for Blood Research, Boston, MA 02115, USA.
Mol Med Today. 1998 Jul;4(7):300-4. doi: 10.1016/s1357-4310(98)01268-4.
Patients with Wiskott-Aldrich syndrome show various defects in the normal function of platelets and lymphocytes. The recent identification of the gene responsible for this syndrome has led to a surge of studies aimed at solving the puzzle posed by the varied phenotype observed in this disease. It is now known that WASP, the protein product of this gene, can interact with a large number of other proteins known to be involved in the regulation of signal transduction and cytoskeletal organization. Thus, WASP appears to integrate these two basic and fundamental cellular mechanisms. Several groups are now focusing on understanding the function of WASP in detail, and translating this new knowledge into improved therapies.
患有威斯科特-奥尔德里奇综合征的患者在血小板和淋巴细胞的正常功能方面表现出各种缺陷。最近对导致该综合征的基因的鉴定引发了大量研究，旨在解决这种疾病中观察到的多样表型所带来的谜团。现在已知，该基因的蛋白质产物WASP能与大量已知参与信号转导调节和细胞骨架组织的其他蛋白质相互作用。因此，WASP似乎整合了这两种基本且重要的细胞机制。目前有几个研究小组正专注于详细了解WASP的功能，并将这一新知识转化为改进的治疗方法。
