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Understanding Ocular Inflammation in Eyes Treated With Intravitreal Gene Therapy

Understanding Ocular Inflammation in Eyes Treated With Intravitreal Gene Therapy Outcomes in Patients With Leber Hereditary Optic Neuropathy Treated With Intravitreal rAAV2/2-ND4 Original Investigation Research Invited Commentary Understanding Ocular Inflammation in Eyes Treated With Intravitreal Gene Therapy Jacque L. Duncan, MD Inherited eye diseases typically cause progressive, irreversible treated. The dose most commonly associated with inflammation vision loss, with limited effective treatments. Recently, a trial of was 9 × 10 vg, with 6 of 6 patients experiencing anterior cham- the first US Food and Drug Administration–approved gene ber inflammation and 5 of 6 demonstrating vitreous inflamma- therapyforinheritedeyediseasedemonstratedthesafetyofgene tion,suggestingthatthelowestdosewasassociatedwiththelow- therapy. Diseases that affect est and mildest inflammation among the patients studied. The the inner retinal ganglion cells, patient who had the most severe anterior and intermediate in- Related article page 399 such as Leber hereditary optic flammation also had the highest baseline and maximum IgG and neuropathy,maybetargetedusinganintravitrealratherthansub- neutralizingantibodylevelsandwas1ofonly2patientswithposi- retinal injection. Vectors that can be delivered intravitreally are tive cellular response, whereas another patient, who also had being developed to target the outer retina, which may cause less positive IgG, neutralizing antibody, and cellular responses after photoreceptor damage but may have a greater risk of systemic injection, did not experience any ocular inflammation. The re- exposure to the viral vector used http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA Ophthalmology American Medical Association

Understanding Ocular Inflammation in Eyes Treated With Intravitreal Gene Therapy

JAMA Ophthalmology , Volume 137 (4) – Apr 7, 2019

Understanding Ocular Inflammation in Eyes Treated With Intravitreal Gene Therapy

Abstract

Outcomes in Patients With Leber Hereditary Optic Neuropathy Treated With Intravitreal rAAV2/2-ND4 Original Investigation Research Invited Commentary Understanding Ocular Inflammation in Eyes Treated With Intravitreal Gene Therapy Jacque L. Duncan, MD Inherited eye diseases typically cause progressive, irreversible treated. The dose most commonly associated with inflammation vision loss, with limited effective treatments. Recently, a trial of was 9 × 10 vg, with 6 of 6 patients...
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Publisher
American Medical Association
Copyright
Copyright 2019 American Medical Association. All Rights Reserved.
ISSN
2168-6165
eISSN
2168-6173
DOI
10.1001/jamaophthalmol.2018.6915
Publisher site
See Article on Publisher Site

Abstract

Outcomes in Patients With Leber Hereditary Optic Neuropathy Treated With Intravitreal rAAV2/2-ND4 Original Investigation Research Invited Commentary Understanding Ocular Inflammation in Eyes Treated With Intravitreal Gene Therapy Jacque L. Duncan, MD Inherited eye diseases typically cause progressive, irreversible treated. The dose most commonly associated with inflammation vision loss, with limited effective treatments. Recently, a trial of was 9 × 10 vg, with 6 of 6 patients experiencing anterior cham- the first US Food and Drug Administration–approved gene ber inflammation and 5 of 6 demonstrating vitreous inflamma- therapyforinheritedeyediseasedemonstratedthesafetyofgene tion,suggestingthatthelowestdosewasassociatedwiththelow- therapy. Diseases that affect est and mildest inflammation among the patients studied. The the inner retinal ganglion cells, patient who had the most severe anterior and intermediate in- Related article page 399 such as Leber hereditary optic flammation also had the highest baseline and maximum IgG and neuropathy,maybetargetedusinganintravitrealratherthansub- neutralizingantibodylevelsandwas1ofonly2patientswithposi- retinal injection. Vectors that can be delivered intravitreally are tive cellular response, whereas another patient, who also had being developed to target the outer retina, which may cause less positive IgG, neutralizing antibody, and cellular responses after photoreceptor damage but may have a greater risk of systemic injection, did not experience any ocular inflammation. The re- exposure to the viral vector used

Journal

JAMA OphthalmologyAmerican Medical Association

Published: Apr 7, 2019

References