Inherited eye diseases typically cause progressive, irreversible vision loss, with limited effective treatments. Recently, a trial of the first US Food and Drug Administration–approved gene therapy for inherited eye disease demonstrated the safety of gene therapy.1 Diseases that affect the inner retinal ganglion cells, such as Leber hereditary optic neuropathy, may be targeted using an intravitreal rather than subretinal injection. Vectors that can be delivered intravitreally are being developed to target the outer retina, which may cause less photoreceptor damage2 but may have a greater risk of systemic exposure to the viral vector used to deliver the genetic material with accompanying inflammation.3
Duncan JL. Understanding Ocular Inflammation in Eyes Treated With Intravitreal Gene Therapy. JAMA Ophthalmol. 2019;137(4):407. doi:10.1001/jamaophthalmol.2018.6915
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