A U.S. research team has developed a novel gene therapy for Leber Congenital Amaurosis (LCA), a rare form of childhood blindness. Unlike conventional AAV-based gene augmentation, this approach utilizes RNA editing technology to directly correct mutations in the CEP290 gene. In preclinical animal models, the therapy demonstrated restoration of retinal function.
The platform may enable targeting of large genes previously inaccessible to standard gene therapy, offering potential applicability to other inherited retinal diseases and neurological disorders. Human clinical trials are anticipated as the next development stage.
Source: Drug Discovery News (June 13, 2025), Article Link
【My Thoughts】
RNA editing for gene correction provides a promising new option complementing current AAV-based gene therapy strategies. The ability to address larger gene targets and expand into broader retinal and neurological indications is particularly noteworthy. Moving forward, clinical development will need to carefully evaluate safety, durability, and potential immune responses.
