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Absence of lenadogene nolparvovec DNA in a brain tumor biopsy from a patient in the REVERSE clinical study, a case report

BACKGROUND: Leber Hereditary Optic Neuropathy (LHON) is a rare, maternally-inherited mitochondrial disease that primarily affects retinal ganglion cells (RGCs) and their axons in the optic nerve, leading to irreversible, bilateral severe vision loss. Lenadogene nolparvovec gene therapy was developed...

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Detalles Bibliográficos
Autores principales: Newman, Nancy J., Schniederjan, Matthew, Mendoza, Pia R., Calkins, David J., Yu-Wai-Man, Patrick, Biousse, Valérie, Carelli, Valerio, Taiel, Magali, Rugiero, Francois, Singh, Pramila, Rogue, Alexandra, Sahel, José-Alain, Ancian, Philippe
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9277876/
https://www.ncbi.nlm.nih.gov/pubmed/35820885
http://dx.doi.org/10.1186/s12883-022-02787-y