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  1. Article

    Open Access

    Expression and processing of mature human frataxin after gene therapy in mice

    Friedreich’s ataxia is a degenerative and progressive multisystem disorder caused by mutations in the highly conserved frataxin (FXN) gene that results in FXN protein deficiency and mitochondrial dysfunction. ...

    Teerapat Rojsajjakul, Nithya Selvan, Bishnu De, Jonathan B. Rosenberg… in Scientific Reports (2024)

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  2. Article

    Open Access

    Validation of Anti-Adeno Associated Virus Serotype rh10 (AAVrh.10) Total and Neutralizing Antibody Immunogenicity Assays

    Immunogenicity assessment of Adeno-Associated Virus (AAV) vectors is a critical part of gene therapy drug development. Whether the assays are used for inclusion/exclusion criteria or to monitor the safety and ...

    Elizabeth Butala-Flores, Thien Nguyen, Nithya Selvan… in Pharmaceutical Research (2023)

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  3. No Access

    Article

    A mutant O-GlcNAcase enriches Drosophila developmental regulators

    An inactive mutant of a bacterial O-GlcNAc hydrolase was used as an affinity reagent to enrich O-GlcNAc-modified proteins from Drosophila embryos and led to the identification, by MS–proteomics, of O-GlcNAcylated...

    Nithya Selvan, Ritchie Williamson, Daniel Mariappa… in Nature Chemical Biology (2017)