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Nature Communications|June 19, 2026
Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome modelsClaudiu A Cozmescu, Mina Nazari, Loukia Touramanidou, et al.
Scientific Reports|February 9, 2020
Generation of light-producing somatic-transgenic mice using adeno-associated virus vectorsRajvinder Karda, Ahad A Rahim, Andrew M S Wong, et al.
Nature Communications|August 31, 2018
Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transferJulien Baruteau, Dany P Perocheau, Joanna Hanley, et al.
Biorxiv : the Preprint Server for Biology|November 28, 2023
Creation of de novo cryptic splicing for ALS/FTD precision medicineOscar G Wilkins, Max Z Y J Chien, Josette J Wlaschin, et al.
Nature Medicine|January 16, 2026
Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapySarah Buddle, Li-An K Brown, Sofia Morfopoulou, et al.
Science (New York, N.Y.)|October 3, 2024
Creation of de novo cryptic splicing for ALS and FTD precision medicineOscar G Wilkins, Max Z Y J Chien, Josette J Wlaschin, et al.
Science Translational Medicine|May 20, 2021
Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonismJoanne Ng, Serena Barral, Carmen De La Fuente Barrigon, et al.
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