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Biodistribution of intravitreal (lenadogene) nolparvovec gene therapy in nonhuman primates

Lenadogene nolparvovec (Lumevoq) gene therapy was developed to treat Leber hereditary optic neuropathy (LHON) caused by the m.11778G > A in MT-ND4 that affects complex I of the mitochondrial respiratory chain. Lenadogene nolparvovec is a replication-defective, single-stranded DNA recombinant aden...

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Detalles Bibliográficos
Autores principales: Calkins, David J., Yu-Wai-Man, Patrick, Newman, Nancy J., Taiel, Magali, Singh, Pramila, Chalmey, Clémentine, Rogue, Alexandra, Carelli, Valerio, Ancian, Philippe, Sahel, José A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8526752/
https://www.ncbi.nlm.nih.gov/pubmed/34729378
http://dx.doi.org/10.1016/j.omtm.2021.09.013