蛋白酪氨酸磷酸酶1B：雷特综合征的一个新治疗靶点。
PTP1B: a new therapeutic target for Rett syndrome.
作者信息
Tautz Lutz
出版信息
J Clin Invest. 2015 Aug 3;125(8):2931-4. doi: 10.1172/JCI83192. Epub 2015 Jul 27.
Abstract
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.
摘要
瑞特综合征（RTT）是一种X连锁神经发育障碍，其特征是后天获得的认知、社交和运动技能逐渐丧失，并出现自闭症行为。RTT在每10000例存活女婴中约有1例受影响，是女性严重智力迟钝的第二大常见原因，仅次于唐氏综合征。目前，尚无治愈RTT的方法或有效疗法。目前批准的治疗方案仅限于对特定身体和精神残疾的支持性管理。在本期杂志中，克里希南及其同事揭示，蛋白酪氨酸磷酸酶PTP1B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PTP1B有潜力成为治疗RTT的可行治疗策略。
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