Eye (London, England)۲۰۲۵نمایه شده در PubMed
Libin Jiang, Sitong Guo, Bin Li, Su Xiao, Fei Wang, Wenbin Wei
چکیده
This study aimed to assess the safety and efficacy of a rAAV2 carrying normal ND4 (rAAV2-ND4) (NR082) in individuals with visual loss due to LHON carrying the m.11778G>A mutation. Additionally, it aimed to determine a safe dose of NR082 for intravitreal injection. This was a single-arm, open-label,…
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DOI: 10.1038/s41433-025-03972-2