Detalhe da pesquisa
1.
Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy.
Brain
; 146(4): 1328-1341, 2023 04 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-36350566
2.
Ocular stress enhances contralateral transfer of lenadogene nolparvovec gene therapy through astrocyte networks.
Mol Ther
; 31(7): 2005-2013, 2023 07 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-37016579
3.
Absence of lenadogene nolparvovec DNA in a brain tumor biopsy from a patient in the REVERSE clinical study, a case report.
BMC Neurol
; 22(1): 257, 2022 Jul 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-35820885
4.
The Impact of Leber Hereditary Optic Neuropathy on the Quality of Life of Patients and Their Relatives: A Qualitative Study.
J Neuroophthalmol
; 42(3): 316-322, 2022 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35483081
5.
Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset.
Ophthalmology
; 128(5): 649-660, 2021 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33451738
6.
Long-Term Follow-Up After Unilateral Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy: The RESTORE Study.
J Neuroophthalmol
; 41(3): 309-315, 2021 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34415265
7.
Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies.
J Neuroophthalmol
; 41(3): 298-308, 2021 Sep 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34310464
8.
Visual Outcomes in Leber Hereditary Optic Neuropathy Patients With the m.11778G>A (MTND4) Mitochondrial DNA Mutation.
J Neuroophthalmol
; 40(4): 547-557, 2020 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-32969847
9.
Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy.
Am J Ophthalmol
; 249: 108-125, 2023 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-36496192
10.
Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation.
Ophthalmol Ther
; 12(1): 401-429, 2023 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-36449262
11.
Natural history of patients with Leber hereditary optic neuropathy-results from the REALITY study.
Eye (Lond)
; 36(4): 818-826, 2022 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33911213
12.
Biodistribution of intravitreal lenadogene nolparvovec gene therapy in nonhuman primates.
Mol Ther Methods Clin Dev
; 23: 307-318, 2021 Dec 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34729378
13.
Safety of Intravitreal Gene Therapy for Treatment of Subjects with Leber Hereditary Optic Neuropathy due to Mutations in the Mitochondrial ND4 Gene: The REVEAL Study.
BioDrugs
; 35(2): 201-214, 2021 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-33566264
14.
Partial recovery of visual function in a blind patient after optogenetic therapy.
Nat Med
; 27(7): 1223-1229, 2021 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-34031601
15.
Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison.
Front Neurol
; 12: 662838, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34108929
16.
Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy.
Sci Transl Med
; 12(573)2020 12 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-33298565
17.
Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect.
Nat Med
; 25(2): 225-228, 2019 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-30559420
18.
[Leber hereditary optic neuropathy: Bilateral improvement of visual acuity following gene therapy by unilateral injection]. / Neuropathie optique héréditaire de Leber - Amélioration bilatérale de la vision après thérapie génique par injection unilatérale.
Med Sci (Paris)
; 37(8-9): 712-715, 2021.
Artigo
em Francês
| MEDLINE | ID: mdl-34491178
19.
Gene Therapies for the Treatment of Leber Hereditary Optic Neuropathy.
Int Ophthalmol Clin
; 61(4): 195-208, 2021 10 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34584057