TY  - JOUR
AU  - Vignal-ClermoḤ, Catherine
AU  - Yu-Wai-Man, Patrick
AU  - Newman, Nancy J
AU  - Carelli, Valerio
AU  - Moster, Mark L
AU  - Biousse, Valerie
AU  - Subramanian, Prem S
AU  - Wang, An-Guor
AU  - Donahue, Sean P
AU  - Leroy, Bart P
AU  - Sadun, Alfredo A
AU  - Klopstock, Thomas
AU  - Sergott, Robert C
AU  - Fernández, Gema Rebolleda
AU  - Chwalisz, Bart K
AU  - Banik, Rudrani
AU  - Taiel, Magali
AU  - Roux, Michel
AU  - Sahel, José-Alain
TI  - Safety of lenadogene nolparvovec gene therapy over 5 years in 189 patients with Leber hereditary optic neuropathy.
JO  - American journal of ophthalmology
VL  - 249
SN  - 0002-9394
CY  - New York, NY
PB  - Elsevier Science
M1  - DZNE-2023-00034
SP  - 108 - 125
PY  - 2023
AB  -  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
KW  - Dependovirus
KW  - Humans
KW  - Optic Atrophy, Hereditary, Leber: drug therapy
KW  - Optic Atrophy, Hereditary, Leber: genetics
KW  - Genetic Vectors
KW  - Parvovirinae: genetics
KW  - Genetic Therapy
KW  - Inflammation: etiology
KW  - Leber hereditary optic neuropathy (Other)
KW  - intravitreal gene therapy (Other)
KW  - safety (Other)
LB  - PUB:(DE-HGF)16
C6  - pmid:36496192
DO  - DOI:10.1016/j.ajo.2022.11.026
UR  - https://pub.dzne.de/record/169155
ER  -