PTP1B: a new therapeutic target for Rett syndrome.
PTP1B: a new therapeutic target for Rett syndrome.
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PTP1B：雷特综合征的新治疗靶点。
DOI：
10.1172/jci83192
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发表时间：
2015
期刊：
影响因子：
--
通讯作者：
Tautz,Lutz
中科院分区：
文献类型：
--
作者：
Tautz,Lutz
Rett综合征（RTT）是一种X连锁神经发育障碍，其特征在于获得性认知、社交和运动技能的连续丧失以及自闭症行为的发展。RTT影响约1/10，000活产女婴，是仅次于唐氏综合征的女性严重智力迟钝的第二大常见原因。目前，RTT没有治愈或有效的治疗方法。核准的治疗方案目前仅限于对特定身体和精神残疾的支持性管理。在这个问题上，Krishnan及其同事揭示了蛋白酪氨酸磷酸酶PTP 1B在RTT患者和小鼠模型中上调，并提供了强有力的证据，表明靶向PTP 1B具有作为治疗RTT的可行治疗策略的潜力。
Rett syndrome (RTT) is an X-linked neurodevelopmental disorder that is characterized by successive loss of acquired cognitive, social, and motor skills and development of autistic behavior. RTT affects approximately 1 in 10,000 live female births and is the second most common cause of severe mental retardation in females, after Down syndrome. Currently, there is no cure or effective therapy for RTT. Approved treatment regimens are presently limited to supportive management of specific physical and mental disabilities. In this issue, Krishnan and colleagues reveal that the protein tyrosine phosphatase PTP1B is upregulated in patients with RTT and in murine models and provide strong evidence that targeting PTP1B has potential as a viable therapeutic strategy for the treatment of RTT.