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 adeno-a...

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Bibliografiske detaljer

Principais autores:	David J. Calkins, Patrick Yu-Wai-Man, Nancy J. Newman, Magali Taiel, Pramila Singh, Clémentine Chalmey, Alexandra Rogue, Valerio Carelli, Philippe Ancian, José A. Sahel
Format:	Artigo
Sprog:	Inglês
Udgivet:	Elsevier 2021-12-01
Serier:	Molecular Therapy: Methods & Clinical Development
Fag:	viral vector transduction recombinant adeno-associated virus vector 2 serotype 2 ND4 qPCR assay biodistribution
Online adgang:	http://www.sciencedirect.com/science/article/pii/S2329050121001510
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