%0 Journal Article
%A Vignal-ClermoḤ, Catherine
%A Yu-Wai-Man, Patrick
%A Newman, Nancy J
%A Carelli, Valerio
%A Moster, Mark L
%A Biousse, Valerie
%A Subramanian, Prem S
%A Wang, An-Guor
%A Donahue, Sean P
%A Leroy, Bart P
%A Sadun, Alfredo A
%A Klopstock, Thomas
%A Sergott, Robert C
%A Fernández, Gema Rebolleda
%A Chwalisz, Bart K
%A Banik, Rudrani
%A Taiel, Magali
%A Roux, Michel
%A Sahel, José-Alain
%T Safety of lenadogene nolparvovec gene therapy over 5 years in 189 patients with Leber hereditary optic neuropathy.
%J American journal of ophthalmology
%V 249
%@ 0002-9394
%C New York, NY
%I Elsevier Science
%M DZNE-2023-00034
%P 108 - 125
%D 2023
%X  To evaluate the safety profile of lenadogene nolparvovec (Lumevoq) in patients with Leber hereditary optic neuropathy.Pooled analysis of safety data from 5 clinical studies.A total of 189 patients received single unilateral or bilateral intravitreal injections of a recombinant adeno-associated virus 2 (rAAV2/2) vector encoding the human wild-type ND4 gene. Adverse events (AEs) were collected throughout the studies, up to 5 years. Intraocular inflammation and increased intraocular pressure (IOP) were ocular AEs of special interest. Other assessments included ocular examinations, vector bio-dissemination, and systemic immune responses against rAAV2/2.Almost all patients (95.2
%K Dependovirus
%K Humans
%K Optic Atrophy, Hereditary, Leber: drug therapy
%K Optic Atrophy, Hereditary, Leber: genetics
%K Genetic Vectors
%K Parvovirinae: genetics
%K Genetic Therapy
%K Inflammation: etiology
%K Leber hereditary optic neuropathy (Other)
%K intravitreal gene therapy (Other)
%K safety (Other)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:36496192
%R 10.1016/j.ajo.2022.11.026
%U https://pub.dzne.de/record/169155