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Scientists study rare blindness to pave way for gene therapy

NCT ID NCT04639635

First seen Jan 11, 2026 · Last updated May 22, 2026 · Updated 24 times

Summary

This study looks at a rare form of retinitis pigmentosa caused by changes in the CNGB1 gene. Researchers want to understand how vision loss progresses over 3 years in 20 people. The goal is to find the best tests to measure the disease, so they can use them in a future gene therapy trial. This study does not offer treatment, but it is an important step toward developing one.

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Contacts and locations

Locations

  • Dr. Stephen H. Tsang

    New York, New York, 10032, United States

  • Eberhard Karls University Tubingen

    Tübingen, Germany

  • Institut de la Vision/Centre de maladies rares du Centre Hospitalier National Ophtalmologique des Quinze-Vingts

    Paris, France

  • Klinikum der Universität München University Eye Hospital, Ludwig-Maximilians-University (LMU) Munich

    München, Bavaria, 80336, Germany

  • Moorfields Eye Hospital NHS Foundation Trust

    London, United Kingdom

  • Wills Eye Hospital

    Philadelphia, Pennsylvania, 19107, United States

Conditions

Explore the condition pages connected to this study.