Gene Therapy

Patients with pathogenic variants experience lower VA tied to length of IS/OS junction.

Investigators reviewed visual acuity, multi-modal retinal imaging, electroretinography, family history.

Study examines use of fluocinolone acetonide for treating DME in patients.

This ability would unlock possibilities for repairing tissue damaged by disease

Intravitreal gene therapy continues to be well tolerated and shows robust efficacy

In study, treatment demonstrates promising biologic activity, safety profile

Patients underwent short-course, perioperative systemic immunosuppression prior to procedure

Investigators find that nanoparticles deliver gene therapy successfully in mice, rats.

Investigators find that nanoparticles deliver gene therapy successfully in mice, rats.