Ocular Post-mortem Analyses with Histopathological and Molecular Assessments in Leber Hereditary Optic Neuropathy Following AAV2 Gene Therapy (P5-11.025)
To report post-mortem ocular analyses in two patients with Leber hereditary optic neuropathy (LHON) who received lenadogene nolparvovec (LN) gene therapy.
Authors
- Valérie Biousse (ORCID: https://orcid.org/0000-0002-3594-850X)
- Fred N. Ross‐Cisneros
- Philippe Ancian (ORCID: https://orcid.org/0000-0003-0905-4745)
- Leonardo Caporali (ORCID: https://orcid.org/0000-0002-0666-4380)
- Magali Taiel (ORCID: https://orcid.org/0000-0003-1139-5178)
- Nancy J. Newman (ORCID: https://orcid.org/0000-0003-4898-7839)
- Alfredo A. Sadun (ORCID: https://orcid.org/0009-0005-5475-761X)
- Valério Carelli (ORCID: https://orcid.org/0000-0003-4923-6404)
- Elisa Boschetti (ORCID: https://orcid.org/0000-0003-4503-2770)
- Lindreth DuBois
- Henry Liu (ORCID: https://orcid.org/0000-0003-2730-607X)
Institutions
- Emory University (US)
- Doheny Eye Institute (US)
- GenSight Biologics (France) (FR)
- Charles River Laboratories (Germany) (DE)
- University of Bologna (IT)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000208980
- Citations
- 1
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.81