Is Gene Therapy the Next Big Step in Vision Loss Treatment?

Retinal diseases, which affect the eye's light-sensitive layer, are a leading cause of vision loss and blindness. Gene therapy offers hope for treating these conditions, with recent advances potentially transforming the field of eye health. However, significant challenges remain, as this Special Feature explores.

Gene therapy involves introducing DNA or RNA into cells to replace defective or missing genes and halt disease progression. While early efforts have successfully achieved therapeutic gene expression in target tissues, they have also led to serious side effects. These include overactive immune responses, unintended gene insertions that activate cancer-related genes, and, in some cases, fatal outcomes.

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