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
Recruiting
99 years or below
All
Phase 2
10 participants needed
1 Location

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 or below
  • Gender: All


Updated on 04 Aug 2024. Study ID: 842816

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