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| Home > Publications Database > Safety of lenadogene nolparvovec gene therapy over 5 years in 189 patients with Leber hereditary optic neuropathy. > print |
| 001 | 169155 | ||
| 005 | 20250127111032.0 | ||
| 024 | 7 | _ | |a 10.1016/j.ajo.2022.11.026 |2 doi |
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| 037 | _ | _ | |a DZNE-2023-00034 |
| 041 | _ | _ | |a English |
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| 100 | 1 | _ | |a Vignal-ClermoḤ, Catherine |b 0 |
| 245 | _ | _ | |a Safety of lenadogene nolparvovec gene therapy over 5 years in 189 patients with Leber hereditary optic neuropathy. |
| 260 | _ | _ | |a New York, NY |c 2023 |b Elsevier Science |
| 336 | 7 | _ | |a article |2 DRIVER |
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| 336 | 7 | _ | |a Journal Article |b journal |m journal |0 PUB:(DE-HGF)16 |s 1680248493_15779 |2 PUB:(DE-HGF) |
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| 336 | 7 | _ | |a Journal Article |0 0 |2 EndNote |
| 520 | _ | _ | |a 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%) received 9 × 1010 viral genomes and 87.8% had at least 2 years of follow-up. Most patients (75.1%) experienced at least one systemic AE, but systemic treatment-related AEs occurred in 3 patients; none were serious. Intraocular inflammation was reported in 75.6% of lenadogene nolparvovec-treated eyes. Almost all intraocular inflammations occurred in the anterior chamber (58.8%) or in the vitreous (40.3%), and were of mild (90.3%) or moderate (8.8%) intensity; most resolved with topical corticosteroids alone. All IOP increases were mild to moderate in intensity. No AE led to study discontinuation. Bio-dissemination of lenadogene nolparvovec and systemic immune response were limited. The safety profile was comparable for patients treated bilaterally and unilaterally.Lenadogene nolparvovec had a good overall safety profile with excellent systemic tolerability, consistent with limited bio-dissemination. The product was well tolerated, with mostly mild ocular side effects responsive to conventional ophthalmologic treatments. |
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| 650 | _ | 7 | |a Leber hereditary optic neuropathy |2 Other |
| 650 | _ | 7 | |a intravitreal gene therapy |2 Other |
| 650 | _ | 7 | |a safety |2 Other |
| 650 | _ | 2 | |a Dependovirus |2 MeSH |
| 650 | _ | 2 | |a Humans |2 MeSH |
| 650 | _ | 2 | |a Optic Atrophy, Hereditary, Leber: drug therapy |2 MeSH |
| 650 | _ | 2 | |a Optic Atrophy, Hereditary, Leber: genetics |2 MeSH |
| 650 | _ | 2 | |a Genetic Vectors |2 MeSH |
| 650 | _ | 2 | |a Parvovirinae: genetics |2 MeSH |
| 650 | _ | 2 | |a Genetic Therapy |2 MeSH |
| 650 | _ | 2 | |a Inflammation: etiology |2 MeSH |
| 700 | 1 | _ | |a Yu-Wai-Man, Patrick |b 1 |
| 700 | 1 | _ | |a Newman, Nancy J |b 2 |
| 700 | 1 | _ | |a Carelli, Valerio |b 3 |
| 700 | 1 | _ | |a Moster, Mark L |b 4 |
| 700 | 1 | _ | |a Biousse, Valerie |b 5 |
| 700 | 1 | _ | |a Subramanian, Prem S |b 6 |
| 700 | 1 | _ | |a Wang, An-Guor |0 P:(DE-HGF)0 |b 7 |
| 700 | 1 | _ | |a Donahue, Sean P |b 8 |
| 700 | 1 | _ | |a Leroy, Bart P |b 9 |
| 700 | 1 | _ | |a Sadun, Alfredo A |b 10 |
| 700 | 1 | _ | |a Klopstock, Thomas |0 P:(DE-2719)2810704 |b 11 |u dzne |
| 700 | 1 | _ | |a Sergott, Robert C |b 12 |
| 700 | 1 | _ | |a Fernández, Gema Rebolleda |b 13 |
| 700 | 1 | _ | |a Chwalisz, Bart K |b 14 |
| 700 | 1 | _ | |a Banik, Rudrani |b 15 |
| 700 | 1 | _ | |a Taiel, Magali |b 16 |
| 700 | 1 | _ | |a Roux, Michel |b 17 |
| 700 | 1 | _ | |a Sahel, José-Alain |b 18 |
| 700 | 1 | _ | |a Group, LHON Study |b 19 |e Collaboration Author |
| 773 | _ | _ | |a 10.1016/j.ajo.2022.11.026 |g p. S0002939422004640 |0 PERI:(DE-600)2019600-3 |p 108 - 125 |t American journal of ophthalmology |v 249 |y 2023 |x 0002-9394 |
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