| FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia I Manoli, JR Sysol, MW Epping, L Li, C Wang, JL Sloan, A Pass, J Gagné, ... JCI insight 3 (23), 2018 | 58 | 2018 |
| Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery L Garcia, C Beley, T Voit, RM Kotin, L Li US Patent 9,598,703, 2017 | 51 | 2017 |
| Production and characterization of novel recombinant adeno-associated virus replicative-form genomes: a eukaryotic source of DNA for gene transfer L Li, EK Dimitriadis, Y Yang, J Li, Z Yuan, C Qiao, C Beley, RH Smith, ... PLoS One 8 (8), e69879, 2013 | 50 | 2013 |
| Development of recombinant adeno-associated virus vectors carrying small interfering RNA (shHec1)-mediated depletion of kinetochore Hec1 protein in tumor cells L Li, L Yang, DA Scudiero, SA Miller, ZX Yu, PT Stukenberg, ... Gene therapy 14 (10), 814-827, 2007 | 39 | 2007 |
| MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy IM Barbash, S Cecchini, AZ Faranesh, T Virag, L Li, Y Yang, RF Hoyt, ... Gene therapy 20 (3), 274-282, 2013 | 36 | 2013 |
| Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene delivery JS Senac, RJ Chandler, JR Sysol, L Li, CP Venditti Gene therapy 19 (4), 385-391, 2012 | 32 | 2012 |
| ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia PO Ilyinskii, AM Michaud, GL Rizzo, CJ Roy, SS Leung, SL Elkins, ... Molecular Therapy-Methods & Clinical Development 22, 279-292, 2021 | 27 | 2021 |
| The DNA minor groove binding agents Hoechst 33258 and 33342 enhance recombinant adeno‐associated virus (rAAV) transgene expression L Li, L Yang, RM Kotin The Journal of Gene Medicine: A cross‐disciplinary journal for research on …, 2005 | 19 | 2005 |
| Systemic gene therapy using an AAV44. 9 vector rescues a neonatal lethal mouse model of propionic acidemia RJ Chandler, G Di Pasquale, EY Choi, D Chang, SN Smith, JL Sloan, ... Molecular Therapy-Methods & Clinical Development 30, 181-190, 2023 | 3 | 2023 |
| Anc80 and AAV8 vectors mediate equivalent long-term hepatic correction of methylmalonyl-coa mutase deficiency in a murine model of methymalonic acidemia (MMA) L Li, RJ Chandler, E Andres-Mateos, R Xiao, P Ilyinskii, ... MOLECULAR THERAPY 26 (5), 450-450, 2018 | 3 | 2018 |
| Muscle targeted transgene expression rescues the lethal phenotype of Mut knockout mice I Manoli, JR Sysol, L Li, RJ Chandler, J Senac, V Hoffmann, P Zerfas, ... MOLECULAR GENETICS AND METABOLISM 102 (3), 248-248, 2011 | 3 | 2011 |
| Combination of an engineered AAV vector Anc80 and tolerogenic nanoparticles encapsulating rapamycin enables efficient transgene expression in mice with pre-existing … PO Ilyinskii, C Roy, A Michaud, L Li, N DeVita, G Rizzo, S Elkins, ... HUMAN GENE THERAPY 29 (12), A168-A168, 2018 | 2 | 2018 |
| Anc80 and SVP Rapamycin: A novel approach to AAV gene therapy for methylmalonic acidemia RJ Chandler, L Li, E Andres-Mateos, R Xiao, P Ilyinski, KT Kishimoto, ... HUMAN GENE THERAPY, MARY ANN LIEBERT 28, 2017-01, 2017 | 2 | 2017 |
| ImmTOR (TM) Tolerogenic Nanoparticles Enhance Transgene Expression after Both Initial and Repeat Dosing in a Mouse Model of Methylmalonic Acidemia Treated with an Anc80 AAV Vector PO Ilyinskii, C Roy, A Michaud, G Rizzo, SS Leung, S Elkins, T Capela, ... MOLECULAR THERAPY 27 (4), 14-15, 2019 | 1 | 2019 |
| The Combination Therapy of ImmTOR (TM) with AAV Anc80 is Therapeutic, Safe, and Repeatable in Mice with Methylmalonic Acidemia, and Compatible with the Low Seroprevalence of … L Li, RJ Chandler, SS Leung, S Elkins, P Ilyinskii, A Pass, I Manoli, ... MOLECULAR THERAPY 27 (4), 202-202, 2019 | 1 | 2019 |
| Mechanisms associated with insulin resistance in adenosine A1 receptor deficient mice R Faulhaber-Walter, D Mizel, L Li, S Kim, J Zhang, M Chen, Y Huang, ... Diabetologie und Stoffwechsel 6 (04), P290, 2011 | 1 | 2011 |
| Closed-ended, linear, duplex adenoassociated virus dna, and uses thereof L Li, R Kotin US Patent App. 18/151,803, 2023 | | 2023 |
| Closed-ended, linear, duplex adenoassociated virus DNA, and uses thereof L Li, R Kotin US Patent 11,549,125, 2023 | | 2023 |
| Systemic AAV9 Human Propionyl-CoA Carboxylase, Alpha Subunit (AAV9-hPCCA) Gene Therapy Provides Direct and Sustained Therapeutic Benefits in the Treatment of Propionic Acidemia … L Li, EY Choi, S Smith, E Wagner, C Chen, X Xu, E Ottinger, PJ Brooks, ... MOLECULAR THERAPY 30 (4), 473-473, 2022 | | 2022 |
| ImmTOR (TM) Tolerogenic Nanoparticles Enable Gene Therapy of Mice with Maternally-Transferred Anti-AAV Antibodies in a Mouse Model of Methylmalonic Acidemia PO Ilyinskii, AM Michaud, GL Rizzo, J LePrevost, CJ Roy, SS Leung, ... MOLECULAR THERAPY 28 (4), 577-578, 2020 | | 2020 |
