| Dose-dependent prevention of metabolic and neurologic disease in murine MPS II by ZFN-mediated in vivo genome editing K Laoharawee, RC DeKelver, KM Podetz-Pedersen, M Rohde, S Sproul, ... Molecular Therapy 26 (4), 1127-1136, 2018 | 125 | 2018 |
| ZFN-mediated in vivo genome editing corrects murine hurler syndrome L Ou, RC DeKelver, M Rohde, S Tom, R Radeke, SJS Martin, Y Santiago, ... Molecular Therapy 27 (1), 178-187, 2019 | 85 | 2019 |
| rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis W Shen, S Liu, L Ou Frontiers in immunology 13, 1001263, 2022 | 69 | 2022 |
| High-dose enzyme replacement therapy in murine Hurler syndrome L Ou, T Herzog, BL Koniar, R Gunther, CB Whitley Molecular genetics and metabolism 111 (2), 116-122, 2014 | 62 | 2014 |
| A highly efficacious PS gene editing system corrects metabolic and neurological complications of mucopolysaccharidosis type I LI Ou, MJ Przybilla, O Ahlat, S Kim, P Overn, J Jarnes, MG O’Sullivan, ... Molecular Therapy 28 (6), 1442-1454, 2020 | 55 | 2020 |
| A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases L Ou, MJ Przybilla, AF Tăbăran, P Overn, MG O’Sullivan, X Jiang, R Sidhu, ... Gene therapy 27 (5), 226-236, 2020 | 51 | 2020 |
| Rare disease awareness and perspectives of physicians in China: a questionnaire-based study X Li, X Zhang, S Zhang, Z Lu, J Zhang, J Zhou, B Li, L Ou Orphanet Journal of Rare Diseases 16, 1-9, 2021 | 41 | 2021 |
| Phenotype prediction for mucopolysaccharidosis type I by in silico analysis L Ou, MJ Przybilla, CB Whitley Orphanet journal of rare diseases 12, 1-14, 2017 | 34 | 2017 |
| Comprehensive behavioral and biochemical outcomes of novel murine models of GM1-gangliosidosis and Morquio syndrome type B MJ Przybilla, L Ou, AF Tăbăran, X Jiang, R Sidhu, PJ Kell, DS Ory, ... Molecular genetics and metabolism 126 (2), 139-150, 2019 | 29 | 2019 |
| Standardization of α-L-iduronidase enzyme assay with Michaelis–Menten kinetics L Ou, TL Herzog, CM Wilmot, CB Whitley Molecular genetics and metabolism 111 (2), 113-115, 2014 | 29 | 2014 |
| A versatile toolkit for overcoming AAV immunity X Li, X Wei, J Lin, L Ou Frontiers in Immunology 13, 991832, 2022 | 26 | 2022 |
| RTB lectin-mediated delivery of lysosomal α-l-iduronidase mitigates disease manifestations systemically including the central nervous system L Ou, MJ Przybilla, B Koniar, CB Whitley Molecular genetics and metabolism 123 (2), 105-111, 2018 | 24 | 2018 |
| Is iPS cell the panacea? L Ou, X Wang, F Zou IUBMB life 62 (3), 170-175, 2010 | 21 | 2010 |
| A questionnaire-based study to comprehensively assess the status quo of rare disease patients and care-givers in China X Li, M Liu, J Lin, B Li, X Zhang, S Zhang, Z Lu, J Zhang, J Zhou, L Ou Orphanet Journal of Rare Diseases 16, 1-12, 2021 | 20 | 2021 |
| Metabolomics profiling reveals profound metabolic impairments in mice and patients with Sandhoff disease L Ou, MJ Przybilla, CB Whitley Molecular genetics and metabolism 126 (2), 151-156, 2019 | 20 | 2019 |
| Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I L Ou, MJ Przybilla, BL Koniar, CB Whitley Molecular Genetics and Metabolism Reports, 93-97, 2016 | 19 | 2016 |
| AAV engineering for improving tropism to the central nervous system MS Ghauri, L Ou Biology 12 (2), 186, 2023 | 18 | 2023 |
| Coping strategies, stress, and support needs in caregivers of children with mucopolysaccharidosis A Schadewald, E Kimball, L Ou JIMD Reports, Volume 42, 89-97, 2018 | 18 | 2018 |
| The urgent need to empower rare disease organizations in China: an interview-based study X Li, Z Lu, J Zhang, X Zhang, S Zhang, J Zhou, B Li, L Ou Orphanet Journal of Rare Diseases 15, 282, 2020 | 15 | 2020 |
| Genotype-phenotype correlation of gangliosidosis mutations using in silico tools and homology modeling L Ou, S Kim, CB Whitley, JR Jarnes-Utz Molecular Genetics and Metabolism Reports 20, 100495, 2019 | 15 | 2019 |
