Biodistribution of intravitreal lenadogene nolparvovec gene therapy in nonhuman primates

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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Bibliographic Details
Main Authors: 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: Article
Language:English
Published: Elsevier 2021-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
viral vector
transduction
recombinant adeno-associated virus vector 2 serotype 2
ND4
qPCR assay
biodistribution
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001510

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http://www.sciencedirect.com/science/article/pii/S2329050121001510

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