PTP1B: a new therapeutic target for Rett syndrome.
Level V
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- Record sourced from PubMed, PMID 26214520.
- Also identified by DOI 10.1172/JCI83192 and PMC identifier 4563768.
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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.
Medical subject headings
- Enzyme Inhibitors
- Protein Tyrosine Phosphatase, Non-Receptor Type 1
- Rett Syndrome
- Signal Transduction