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PLOS ONE: an inclusive, peer-reviewed, open-access resource from the PUBLIC LIBRARY OF SCIENCE. Reports of well-performed scientific studies from all disciplines freely available to the whole world.
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The UshStat, an EIAV-based lentiviral gene therapy vector harboring a full length human myosin VIIa expression cassette under the control of a constitutive CMV promoter, restores the α-transducin translocation threshold and reduces the light-dependent photoreceptor degeneration phenotypes in shaker1 mice. These data provided support for the initiation of the first-in-man clinical trial of UshStat in patients with Usher syndrome 1B (ClinicalTrials.gov identifier: NCT01505062) that is currently underway.
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