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    Gene therapy for choroideremia may be on horizon


    New phase II trial

    The new open-label phase II trial will give a single sub-retinal injection of 0.1 mL AAV2-REP1 to one eye only in six men. Safety will be assessed as the primary outcome, with changes in visual field and function as secondary outcomes.

    The initial follow-up period will be 2 years, with further annual follow-up for at least 10 years to test the long-lasting benefit of the therapy.

    Data will continue to be analysed even after the study is complete.

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