Clinical Challenges: Gene Therapy for Inherited Retinal Diseases

Refinements in surgical approach have made subretinal delivery very safe, efficient, expert says

The FDA approval of UF‘s AAV technology in voretigene neparvovec (Luxturna) for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy almost 4 years ago marked a turning point in gene therapy.

Since then, clinicians have witnessed the positive impact that gene therapy has had on the lives of patients and their families.

“Luxturna dramatically improves retinal sensitivity, resulting in improved night vision in most patients and even visual acuity in some patients,” said Mark Pennesi, MD, Ph.D., of the Casey Eye Institute Ophthalmic Genetic Clinic at Oregon Health & Science University in Portland, in an interview.

Read more about Clinical Challenges: Gene Therapy for Inherited Retinal Diseases.