| CRISPR/Cas9-mediated genome editing as a therapeutic approach for Leber congenital amaurosis 10 GX Ruan, E Barry, D Yu, M Lukason, SH Cheng, A Scaria Molecular therapy 25 (2), 331-341, 2017 | 297 | 2017 |
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| Preclinical safety evaluation of AAV2-sFLT01—a gene therapy for age-related macular degeneration TK MacLachlan, M Lukason, M Collins, R Munger, E Isenberger, ... Molecular Therapy 19 (2), 326-334, 2011 | 199 | 2011 |
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| Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule M Lukason, E DuFresne, H Rubin, P Pechan, Q Li, I Kim, S Kiss, C Flaxel, ... Molecular Therapy 19 (2), 260-265, 2011 | 110 | 2011 |
| A concentrated and stable aerosol formulation of cationic lipid: DNA complexes giving high-level gene expression in mouse lung SJ Eastman, MJ Lukason, JD Tousignant, H Murray, MD Lane, ... Human gene therapy 8 (6), 765-773, 1997 | 107 | 1997 |
| EGTA Enhancement of Adenovirus-Mediated Gene Transfer to Mouse Tracheal Epithelium in Vivo Q Chu, JA St. George, M Lukason, SH Cheng, RK Scheule, SJ Eastman Human gene therapy 12 (5), 455-467, 2001 | 84 | 2001 |
| Adenovirus-transduced lung as a portal for delivering α-galactosidase A into systemic circulation for Fabry disease C Li, RJ Ziegler, M Cherry, M Lukason, RJ Desnick, NS Yew, SH Cheng Molecular Therapy 5 (6), 745-754, 2002 | 49 | 2002 |
| Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases MJ Schmidt, A Gupta, C Bednarski, S Gehrig-Giannini, F Richter, C Pitzler, ... Nature communications 12 (1), 4219, 2021 | 44 | 2021 |
| Aerosolization of Cationic Lipid:pDNA Complexes—In Vitro Optimization of Nebulizer Parameters for Human Clinical Studies SJ Eastman, JD Tousignant, MJ Lukason, Q Chu, SH Cheng, RK Scheule Human gene therapy 9 (1), 43-52, 1998 | 41 | 1998 |
| Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain JA Sullivan, LM Stanek, MJ Lukason, J Bu, SR Osmond, EA Barry, ... Gene Therapy 25 (3), 205-219, 2018 | 30 | 2018 |
| Cytotoxic T lymphocyte responses to transgene product, not adeno-associated viral capsid protein, limit transgene expression in mice WM Siders, J Shields, J Kaplan, M Lukason, L Woodworth, S Wadsworth, ... Human gene therapy 20 (1), 11-20, 2009 | 26 | 2009 |
| Engineered capsids for efficient gene delivery to the retina and cornea A Frederick, J Sullivan, L Liu, M Adamowicz, M Lukason, J Raymer, Z Luo, ... Human Gene Therapy 31 (13-14), 756-774, 2020 | 23 | 2020 |
| Adenoviral vector expressing ICP47 inhibits adenovirus-specific cytotoxic T lymphocytes in nonhuman primates A Scaria, JA Sullivan, JAS George, JM Kaplan, MJ Lukason, JE Morris, ... Molecular Therapy 2 (5), 505-514, 2000 | 16 | 2000 |
| biAb Mediated Restoration of the Linkage between Dystroglycan and Laminin-211 as a Therapeutic Approach for α-Dystroglycanopathies N Gumlaw, LM Sevigny, H Zhao, Z Luo, DS Bangari, E Masterjohn, ... Molecular Therapy 28 (2), 664-676, 2020 | 10 | 2020 |
| Ingestion in vivo by alveolar macrophages of perfluorochemical (PFC) liquid correlates with altered pro-inflammatory cytokine release L Bonneau, TH Shaffer, M Lukason, J St George, MR Wolfson, DJ Weiss Am J Respir Crit Care Med 151, 902A, 2000 | 8 | 2000 |
| Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis SL Boye, C O’Riordan, J Morris, M Lukason, D Compton, R Baek, ... Molecular Therapy Methods & Clinical Development 28, 129-145, 2023 | 5 | 2023 |
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