Li-Na Li

Most-cited papers

1. FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia.

   I. Manoli, J. Sysol, M. Epping, Li-Na Li +27 more

   2018JCI Insight66 citations Open access

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2. MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy

   I. Barbash, Sylvain Cecchini, A. Faranesh, T. Virág +8 more

   2012Gene Therapy34 citations Open access

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3. The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup

   P. Brooks, E. Ottinger, D. Portero, Richa Madan Lomash +35 more

   2020Human Gene Therapy33 citations Open access

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4. ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia

   P. Ilyinskii, Alicia M. Michaud, G. Rizzo, C. Roy +12 more

   2021Molecular Therapy: Methods & Clinical Development32 citations Open access

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5. Production and Characterization of Novel Recombinant Adeno-Associated Virus Replicative-Form Genomes: A Eukaryotic Source of DNA for Gene Transfer

   Li-Na Li, E. Dimitriadis, Yu Yang, Juan Li +6 more

   2013PLoS ONE15 citations Open access

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6. Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia

   R. Chandler, G. Di Pasquale, Eun-Young Choi, David Chang +6 more

   2023Molecular Therapy: Methods & Clinical Development9 citations Open access

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7. The DNA minor groove binding agents Hoechst 33258 and 33342 enhance recombinant adeno‐associated virus (rAAV) transgene expression

   Li-Na Li, Linda Yang, R. Kotin

   20059 citations

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8. The DNA minor groove binding agents Hoechst 33258 and 33342 enhance recombinant adeno-associated virus (rAAV) transgene expression.

   Li-Na Li, Linda Yang, R. Kotin

   2004Journal of Gene Medicine9 citations Open access

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9. Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program

   Richa Madan Lomash, Jean Dehdashti, Oleg A. Shchelochkov, R. Chandler +13 more

   2025Human Gene Therapy6 citations Open access

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10. Adeno-Associated Virus Gene Therapy Translation: Lessons from Early Regulatory Meetings

    R. Stan, Richa Madan Lomash, Oleg A. Shchelochkov, I. Manoli +13 more

    2026Human Gene Therapy0 citations Open access

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11. AAV Rep protein-dependent expression of GFP.

    Li-Na Li, K. DimitriadisEmilios, Yu Yang, Juan Li +6 more

    20130 citations Open access

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12. 716. Transducing Efficiency Screening for rAAV2, rAAV1, and rAAV5 in 56 Human Tumor Cell Lines

    Li-Na Li, Linda Yang, D. Scudiero, R. Shoemaker +1 more

    20050 citations Open access

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