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Biodistribution of intravitreal lenadogene nolparvovec gene therapy in nonhuman primates

1 Oct 2021

Abstract excerpt

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-associated virus vector 2 serotype 2, containing a codon-optimized complementary DNA encoding the human wild-type...

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PMID: 34729378
DOI: 10.1016/j.omtm.2021.09.013

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