Profesionales médicos

Dec. 13, 2025

A Mayo Clinic study published in the September 2025 issue of Science Advances explores a novel approach to retinal gene therapy using fibrin hydrogels encapsulating adeno-associated virus 2-green fluorescent protein (AAV2-GFP). The findings suggest a promising, less invasive alternative for the treatment of blinding retinal diseases.

Luxturna was approved by the Food and Drug Administration in 2017 for RPE65-related inherited retinal dystrophy and was the first in vivo gene therapy. While subretinal injection of adeno-associated virus (AAV) is the current standard of care for retinal gene therapy and has restored functional vision to many patients, it can cause localized damage and limits gene expression to the treated area.

"The traditional method for delivering gene therapy in patients with an inherited retinal disease involves surgically injecting the treatment underneath the retina," says Brittni A. Scruggs, M.D., Ph.D., the lead author of the article and a vitreoretinal surgeon at Mayo Clinic in Rochester, Minnesota. "This is a procedure with several known complications associated with this route of administration."

Dr. Scruggs and her colleagues, including Raymond Iezzi Jr., M.D., and Alan D. Marmorstein, Ph.D., in the Retinal Regenerative Medicine Laboratory at Mayo Clinic developed high-concentration fibrin gels with uniform AAV distribution and tested their epiretinal placement.

"With this newly developed, alternative approach, the therapy is placed gently on the surface of the retina within a fibrin hydrogel," says Dr. Marmorstein. "This special gel releases the gene therapy as it degrades within a few days, which offers the potential to treat larger retinal areas with less surgical trauma."

Placing the gene therapy inside a fibrin-based hydrogel directly on top of the retina — rather than underneath — allows the therapy to reach a wide area of retinal cells while using low doses. "In a large animal model, this epiretinal delivery method led to broad gene expression with less inflammation compared to traditional methods," says Dr. Marmorstein.

The study's findings have shown promise for both current and future patients. "This new technique could potentially expand the range of patients who can benefit from gene therapy and make treatment safer," says Dr. Scruggs, "while also preserving — or even enhancing — the excellent results the treatments, such as Luxturna, have already achieved for eligible individuals."

As principal investigators in the Retinal Regenerative Medicine Laboratory at Mayo Clinic, Drs. Scruggs, Iezzi and Marmorstein continue to focus on the optimization of gene therapy and cell-based therapy for improved safety and efficacy in patients with retinal degeneration.

"Our next step is to test this new method head-to-head with existing techniques in models of retinal disease to see if it can further improve visual rescue," says Dr. Scruggs.

For more information

Scruggs BA, et al. Retinal gene therapy using epiretinal AAV-containing fibrin hydrogel implants. Science Advances. 2025;11:eadv7922.

Refer a patient to Mayo Clinic.