McClements, Michelle E.

Codon-optimized RPGR improves stability and efficacy of AAV8 gene therapy in two mouse models of X-linked retinitis pigmentosa

Fischer, M. D., McClements, M. E., Martinez-Fernandez de la Camara, C., Bellingrath, J.-S., Dauletbekov, D., Ramsden, S. C., Hickey, D. G., Barnard, A. R., & MacLaren, R. E. (2017) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 112

An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in Adult Abca4 −/− Mice

McClements, M. E., Barnard, A. R., Singh, M. S., Charbel Issa, P., Jiang, Z., Radu, R. A., & MacLaren, R. E. (2019) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 92

Gene therapy for retinal disease

McClements, M. E., & MacLaren, R. E. (2013) · Translational Research

Retinal Development and DisordersVirus-based gene therapy research

Cited by 80

Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina

Hickey, D. G., Edwards, T. L., Barnard, A. R., Singh, M. S., de Silva, S. R., McClements, M. E., Flannery, J. G., Hankins, M. W., & MacLaren, R. E. (2017) · Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 72

Enhancement of adeno-associated virus-mediated gene therapy using hydroxychloroquine in murine and human tissues

Chandler, L. C., Barnard, A. R., Caddy, S. L., Patrício, M. I., McClements, M. E., Fu, H., Rada, C., MacLaren, R. E., & Xue, K. (2019) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 50

Therapy Approaches for Stargardt Disease

Piotter, E., McClements, M. E., & MacLaren, R. E. (2021) · Biomolecules

Retinal Development and DisordersRetinal Diseases and Treatments

Cited by 49

Immunomodulatory Effects of Hydroxychloroquine and Chloroquine in Viral Infections and Their Potential Application in Retinal Gene Therapy

Chandler, L., Yusuf, I., McClements, M., Barnard, A., MacLaren, R., & Xue, K. (2020) · International Journal of Molecular Sciences

Virus-based gene therapy researchDrug-Induced Ocular Toxicity

Cited by 49

Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina

Chandler, L. C., McClements, M. E., Yusuf, I. H., Martinez-Fernandez de la Camara, C., MacLaren, R. E., & Xue, K. (2021) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 43

Genome-editing strategies for treating human retinal degenerations

Quinn, J., Musa, A., Kantor, A., McClements, M. E., Cehajic-Kapetanovic, J., MacLaren, R. E., & Xue, K. (2021) · Human Gene Therapy

Retinal Development and DisordersCRISPR and Genetic Engineering

Cited by 41

Non-viral delivery of CRISPR/Cas cargo to the retina using nanoparticles: current possibilities, challenges, and limitations

Salman, A., Kantor, A., McClements, M. E., Marfany, G., Trigueros, S., & MacLaren, R. E. (2022) · Pharmaceutics

RNA Interference and Gene DeliveryAdvanced Drug Delivery Systems

Cited by 37

Tropism of AAV Vectors in Photoreceptor-Like Cells of Human iPSC-Derived Retinal Organoids

McClements, M. E., Steward, H., Atkin, W., Goode, E. A., Gándara, C., Chichagova, V., & MacLaren, R. E. (2022) · Translational Vision Science & Technology

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 36

Single-cell transcriptomic analysis of retinal immune regulation and blood-retinal barrier function during experimental autoimmune uveitis

Quinn, J., Salman, A., Paluch, C., Jackson-Wood, M., McClements, M. E., Luo, J., Davis, S. J., Cornall, R. J., MacLaren, R. E., Dendrou, C. A., et al. (2024) · Scientific Reports

Ocular Diseases and Behçet’s SyndromeGlaucoma and retinal disorders

Cited by 32

Assessment of AAV Dual Vector Safety in the Abca4−/− Mouse Model of Stargardt Disease

McClements, M. E., Barnard, A. R., Charbel Issa, P., & MacLaren, R. E. (2020) · Translational Vision Science & Technology

Retinal Development and DisordersVirus-based gene therapy research

Cited by 18

Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration

Fry, L. E., Major, L., Salman, A., McDermott, L. A., Yang, J., King, A. J., McClements, M. E., & MacLaren, R. E. (2025) · Communications Biology

CRISPR and Genetic EngineeringRetinal Development and Disorders

Cited by 15

A Fragmented Adeno-Associated Viral Dual Vector Strategy for Treatment of Diseases Caused by Mutations in Large Genes Leads to Expression of Hybrid Transcripts

McClements, M. E., Charbel Issa, P., Blouin, V., & MacLaren, R. E. (2016) · Journal of Genetic Syndromes & Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 13

Enhancement of adeno-associated virus-mediated gene therapy using hydroxychloroquine in murine and human tissues

Chandler, L. C., Barnard, A. R., Caddy, S. L., Patrício, M. I., McClements, M. E., Fu, H., Rada, C., MacLaren, R. E., & Xue, K. (2023) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 2