USAResearch and clinical trials
Gene therapy improved vision in 3 adults with Leber congenital amaurosis caused by the LCA5 gene
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
In the USA, a gene therapy was tested for congenital blindness due to the LCA5 gene. The early-stage study included 3 adults. The drug was given under the retina in one eye. No serious side effects were seen. Sensitivity to light increased within one month and lasted for one year.
What does it mean for patients and families?
This is a very small, early-stage study. The results can only be confirmed by larger studies. LCA has many different genetic types.
Source
Scientific paper: Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial. Molecular Therapy, 2025. DOI: 10.1016/j.ymthe.2025.06.035 · PubMed 40598770
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Leber congenital amaurosis (LCA)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.