| CRISPR-Cas9 DNA base-editing and prime-editing A Kantor, ME McClements, RE MacLaren International journal of molecular sciences 21 (17), 6240, 2020 | 239 | 2020 |
| Function of human pluripotent stem cell-derived photoreceptor progenitors in blind mice AO Barnea-Cramer, W Wang, SJ Lu, MS Singh, C Luo, H Huo, ... Scientific reports 6 (1), 29784, 2016 | 159 | 2016 |
| Focus: Genome Editing: Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes ME McClements, RE MacLaren The Yale journal of biology and medicine 90 (4), 611, 2017 | 118 | 2017 |
| Codon-optimized RPGR improves stability and efficacy of AAV8 gene therapy in two mouse models of X-linked retinitis pigmentosa MD Fischer, ME McClements, CMF de la Camara, JS Bellingrath, ... Molecular Therapy 25 (8), 1854-1865, 2017 | 118 | 2017 |
| The PROM1 mutation p. R373C causes an autosomal dominant bull's eye maculopathy associated with rod, rod–cone, and macular dystrophy M Michaelides, MC Gaillard, P Escher, L Tiab, M Bedell, FX Borruat, ... Investigative ophthalmology & visual science 51 (9), 4771-4780, 2010 | 112 | 2010 |
| Long-term restoration of visual function in end-stage retinal degeneration using subretinal human melanopsin gene therapy SR De Silva, AR Barnard, S Hughes, SKE Tam, C Martin, MS Singh, ... Proceedings of the National Academy of Sciences 114 (42), 11211-11216, 2017 | 84 | 2017 |
| Gene therapy for retinal disease ME McClements, RE MacLaren Translational Research 161 (4), 241-254, 2013 | 84 | 2013 |
| An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in Adult Abca4−/− Mice ME McClements, AR Barnard, MS Singh, P Charbel Issa, Z Jiang, ... Human gene therapy 30 (5), 590-600, 2019 | 82 | 2019 |
| Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina DG Hickey, TL Edwards, AR Barnard, MS Singh, SR de Silva, ... Gene therapy 24 (12), 787-800, 2017 | 73 | 2017 |
| Inclusion of the woodchuck hepatitis virus posttranscriptional regulatory element enhances AAV2-driven transduction of mouse and human retina MI Patrício, AR Barnard, HO Orlans, ME McClements, RE MacLaren Molecular Therapy-Nucleic Acids 6, 198-208, 2017 | 65 | 2017 |
| Variations in opsin coding sequences cause X-linked cone dysfunction syndrome with myopia and dichromacy M McClements, WIL Davies, M Michaelides, T Young, M Neitz, ... Investigative ophthalmology & visual science 54 (2), 1361-1369, 2013 | 58 | 2013 |
| Clinical and molecular characterization of PROM1-related retinal degeneration J Cehajic-Kapetanovic, J Birtel, ME McClements, ME Shanks, P Clouston, ... JAMA Network Open 2 (6), e195752-e195752, 2019 | 53 | 2019 |
| RNA editing as a therapeutic approach for retinal gene therapy requiring long coding sequences LE Fry, CF Peddle, AR Barnard, ME McClements, RE MacLaren International Journal of Molecular Sciences 21 (3), 777, 2020 | 51 | 2020 |
| Optogenetic gene therapy for the degenerate retina: recent advances ME McClements, F Staurenghi, RE MacLaren, J Cehajic-Kapetanovic Frontiers in neuroscience 14, 570909, 2020 | 48 | 2020 |
| Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells AK Dreismann, ME McClements, AR Barnard, E Orhan, JP Hughes, ... Gene Therapy 28 (5), 265-276, 2021 | 38 | 2021 |
| Immunomodulatory effects of hydroxychloroquine and chloroquine in viral infections and their potential application in retinal gene therapy LC Chandler, IH Yusuf, ME McClements, AR Barnard, RE MacLaren, ... International journal of molecular sciences 21 (14), 4972, 2020 | 38 | 2020 |
| Molecular strategies for RPGR gene therapy J Cehajic Kapetanovic, ME McClements, ... Genes 10 (9), 674, 2019 | 38 | 2019 |
| Therapy approaches for Stargardt disease E Piotter, ME McClements, RE MacLaren Biomolecules 11 (8), 1179, 2021 | 34 | 2021 |
| Enhancement of adeno-associated virus-mediated gene therapy using hydroxychloroquine in murine and human tissues LC Chandler, AR Barnard, SL Caddy, MI Patrício, ME McClements, H Fu, ... Molecular Therapy Methods & Clinical Development 14, 77-89, 2019 | 34 | 2019 |
| A fragmented adeno-associated viral dual vector strategy for treatment of diseases caused by mutations in large genes leads to expression of hybrid transcripts ME McClements, PC Issa, V Blouin, RE MacLaren Journal of genetic syndromes & gene therapy 7 (5), 2016 | 33 | 2016 |
