08/11/2026
A promising step forward for patients with rare inherited blindness. 👁️🧬
University of Iowa Health Care researchers helped lay the foundation for the first-in-human gene therapy study for AIPL1-associated retinal disease—a rare inherited condition that causes severe vision loss in early childhood. The investigational therapy was recently administered to three pediatric patients in the United Kingdom, marking an important milestone in translating decades of laboratory research into potential treatments. The University of Iowa's preclinical research provided the scientific rationale for advancing this therapy to human studies.
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Research conducted at the University of Iowa helped lay the scientific foundation for a first-in-human gene therapy study that recently treated its first three patients in the United Kingdom.The investigational therapy targets retinal degeneration caused by mutations in the BBS10 gene, one of the...