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VGR-R01 Bietti Crystalline Dystrophy (BCD), PI/II

  • Disease Biology

    Bietti Crystalline Dystrophy (BCD) is an autosomal recessive inherited disease caused by loss-of-function mutation of CYP4V2, resulting in lipid deposits on the retina and progressive atrophy of retina and blindness. It is more common in the Chinese and Japanese populations than Caucasians.

  • Signs and Symptoms

    Distinguishing yellow-white crystalline deposits in patient’s retina and cornea. BCD patients always present with vision loss and night blindness between 20 and 40 years of age and eventually progress to legal blindness between the ages of 50 and 60.

  • Epidemiology/Prevalence

    China: 1/20,000
    Global: 1/67,000

  • Available Care/Treatments

    No available treatment.

  • Vitalgen Strategy
    Gene replacement by rAAV8 delivery of a functional cytochrome P450 4v2 (CYP4V2) transgene via sub-retinal injection
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    VGR-R01 Data
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