Kay, Mark A.

Progress and problems with the use of viral vectors for gene therapy

Thomas, C. E., Ehrhardt, A., & Kay, M. A. (2003) · Nature Reviews Genetics

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 2,501

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 2,163

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Nathwani, A. C., Tuddenham, E. G. D., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D. C., Chowdary, P., Riddell, A., Pie, A. J., Harrington, C., et al. (2011) · New England Journal of Medicine

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 1,864

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

Grimm, D., Streetz, K. L., Jopling, C. L., Storm, T. A., Pandey, K., Davis, C. R., Marion, P., Salazar, F., & Kay, M. A. (2006) · Nature

RNA Interference and Gene DeliveryMicroRNA in disease regulation

Cited by 1,677

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Nathwani, A. C., Reiss, U. M., Tuddenham, E. G. D., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., et al. (2014) · New England Journal of Medicine

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 1,273

Robust expansion of human hepatocytes in Fah−/−/Rag2−/−/Il2rg−/− mice

Azuma, H., Paulk, N., Ranade, A., Dorrell, C., Al-Dhalimy, M., Ellis, E., Strom, S., Kay, M. A., Finegold, M., & Grompe, M. (2007) · Nature Biotechnology

Xenotransplantation and immune responseLiver physiology and pathology

Cited by 835

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Manno, C. S., Chew, A. J., Hutchison, S., Larson, P. J., Herzog, R. W., Arruda, V. R., Tai, S. J., Ragni, M. V., Thompson, A., Ozelo, M., et al. (2003) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 749

A nonviral minicircle vector for deriving human iPS cells

Jia, F., Wilson, K. D., Sun, N., Gupta, D. M., Huang, M., Li, Z., Panetta, N. J., Chen, Z. Y., Robbins, R. C., Kay, M. A., et al. (2010) · Nature Methods

Pluripotent Stem Cells ResearchCRISPR and Genetic Engineering

Cited by 734

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

Grimm, D., Lee, J. S., Wang, L., Desai, T., Akache, B., Storm, T. A., & Kay, M. A. (2008) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 696

Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8

Inagaki, K., Fuess, S., Storm, T. A., Gibson, G. A., Mctiernan, C. F., Kay, M. A., & Nakai, H. (2006) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 610

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

Nakai, H., Yant, S. R., Storm, T. A., Fuess, S., Meuse, L., & Kay, M. A. (2001) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 478

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Lisowski, L., Dane, A. P., Chu, K., Zhang, Y., Cunningham, S. C., Wilson, E. M., Nygaard, S., Grompe, M., Alexander, I. E., & Kay, M. A. (2014) · Nature

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 450

Fate tracing of mature hepatocytes in mouse liver homeostasis and regeneration

Malato, Y., Naqvi, S., Schürmann, N., Ng, R., Wang, B., Zape, J., Kay, M. A., Grimm, D., & Willenbring, H. (2011) · Journal of Clinical Investigation

Liver physiology and pathologyTissue Engineering and Regenerative Medicine

Cited by 434

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

Snyder, R. O., Miao, C., Meuse, L., Tubb, J., Donahue, B. A., Lin, H.-F., Stafford, D. W., Patel, S., Thompson, A. R., Nichols, T., et al. (1999) · Nature Medicine

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 409

AAV serotype 2 vectors preferentially integrate into active genes in mice

Nakai, H., Montini, E., Fuess, S., Storm, T. A., Grompe, M., & Kay, M. A. (2003) · Nature Genetics

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 392

Rapid Uncoating of Vector Genomes Is the Key toEfficient Liver Transduction with Pseudotyped Adeno-Associated VirusVectors

Thomas, C. E., Storm, T. A., Huang, Z., & Kay, M. A. (2004) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 358

Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6

Grimm, D., Kay, M. A., & Kleinschmidt, J. A. (2003) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 344

A robust system for production of minicircle DNA vectors

Kay, M. A., He, C.-Y., & Chen, Z.-Y. (2010) · Nature Biotechnology

RNA Interference and Gene DeliveryBacterial Genetics and Biotechnology

Cited by 329

Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk

Vollrath, D., Feng, W., Duncan, J. L., Yasumura, D., D'Cruz, P. M., Chappelow, A., Matthes, M. T., Kay, M. A., & LaVail, M. M. (2001) · Proceedings of the National Academy of Sciences

Retinal Development and DisordersRetinal Diseases and Treatments

Cited by 290

Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors

Narsinh, K. H., Jia, F., Robbins, R. C., Kay, M. A., Longaker, M. T., & Wu, J. C. (2011) · Nature Protocols

Pluripotent Stem Cells ResearchMesenchymal stem cell research

Cited by 214

Human COL7A1 -corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa

Sebastiano, V., Zhen, H. H., Haddad, B., Bashkirova, E., Melo, S. P., Wang, P., Leung, T. L., Siprashvili, Z., Tichy, A., Li, J., et al. (2014) · Science Translational Medicine

Pluripotent Stem Cells ResearchSkin and Cellular Biology Research

Cited by 213

Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver

Grimm, D., Wang, L., Lee, J. S., Schürmann, N., Gu, S., Börner, K., Storm, T. A., & Kay, M. A. (2010) · Journal of Clinical Investigation

RNA Interference and Gene DeliveryVirus-based gene therapy research

Cited by 196

Recruitment of Single-Stranded Recombinant Adeno-Associated Virus Vector Genomes and Intermolecular Recombination Are Responsible for Stable Transduction of Liver In Vivo

Nakai, H., Storm, T. A., & Kay, M. A. (2000) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 194

Large‐scale molecular characterization of adeno‐associated virus vector integration in mouse liver

Nakai, H., Wu, X., Fuess, S., Storm, T. A., Munroe, D., Montini, E., Burgess, S. M., Grompe, M., & Kay, M. A. (2005) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 190