Rett Syndrome: Crossing the Threshold to Clinical Translation.
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ABSTRACT:
SUBMITTER: Katz DM
PROVIDER: S-EPMC4924590 | biostudies-literature | 2016 Feb
REPOSITORIES: biostudies-literature
Publications
Rett Syndrome: Crossing the Threshold to Clinical Translation.
Katz David M DM Bird Adrian A Coenraads Monica M Gray Steven J SJ Menon Debashish U DU Philpot Benjamin D BD Tarquinio Daniel C DC
Trends in neurosciences 20160201 2
Lying at the intersection between neurobiology and epigenetics, Rett syndrome (RTT) has garnered intense interest in recent years, not only from a broad range of academic scientists, but also from the pharmaceutical and biotechnology industries. In addition to the critical need for treatments for this devastating disorder, optimism for developing RTT treatments derives from a unique convergence of factors, including a known monogenic cause, reversibility of symptoms in preclinical models, a stro ...[more]
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