A Multiple-Site Phase 1/2 Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing CNGB3 (rAAV2tYF-PR1.7-hCNGB3) in Patients with Congenital Achromatopsia Caused by Mutations in the CNGB3 Gene

A Multiple-Site  Phase 1/2  Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing CNGB3 (rAAV2tYF-PR1.7-hCNGB3) in Patients with Congenital Achromatopsia Caused by Mutations in the CNGB3 Gene

Brief description of study

Achromatopsia is an inherited retinal disorder characterized by markedly reduced visual acuity, nystagmus, photoaversion (intolerance to bright light), a small central scotoma, eccentric viewing, and complete or severe loss of color discrimination. The genetic mutations of the disease can be established in the majority of individuals. Approximately 50% are caused by mutations in the cone-specific cyclic nucleotide gated channel beta subunit (CNGB3) gene.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    Achromatopsia,inhertited retinal disorder,Eye disorder,reduced vision
  • Age: - 99 Years
  • Gender: All


Updated on 16 Oct 2020. Study ID: 842816

Interested in the study

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