Kay, Mark A.

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

Paulk, N. K., Pekrun, K., Zhu, E., Nygaard, S., Li, B., Xu, J., Chu, K., Leborgne, C., Dane, A. P., Haft, A., et al. (2018) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 161

Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡

Paulk, N. K., Wursthorn, K., Wang, Z., Finegold, M. J., Kay, M. A., & Grompe, M. (2010) · Hepatology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 135

Sustainable correction of junctional epidermolysis bullosa via transposon‐mediated nonviral gene transfer

Ortiz-Urda, S., Lin, Q., Yant, S. R., Keene, D., Kay, M. A., & Khavari, P. A. (2003) · Gene Therapy

Skin and Cellular Biology ResearchVirus-based gene therapy research

Cited by 118

Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype

Grimm, D., Pandey, K., Nakai, H., Storm, T. A., & Kay, M. A. (2006) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 118

Adeno‐associated virus vectors and hematology

Russell, D. W., & Kay, M. A. (1999) · Blood

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 104

Gene therapy for the hemophilias

Kay, M. A., & High, K. (1999) · Proceedings of the National Academy of Sciences

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 96

Helper‐independent and AAV‐ITR‐independent chromosomal integration of double‐stranded linear DNA vectors in mice

Nakai, H., Montini, E., Fuess, S., Storm, T. A., Meuse, L., Finegold, M., Grompe, M., & Kay, M. A. (2003) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 52

AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence

Wang, Z., Lisowski, L., Finegold, M. J., Nakai, H., Kay, M. A., & Grompe, M. (2012) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 44

Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell model

Song, R., Pekrun, K., Khan, T. A., Zhang, F., Paşca, S. P., & Kay, M. A. (2022) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchBarrier Structure and Function Studies

Cited by 36

A 5′ Noncoding Exon Containing Engineered Intron Enhances Transgene Expression from Recombinant AAV Vectors in vivo

Lu, J., Williams, J. A., Luke, J., Zhang, F., Chu, K., & Kay, M. A. (2017) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 26

AAV-mediated genome editing is influenced by the formation of R-loops

Puzzo, F., Crossley, M. P., Goswami, A., Zhang, F., Pekrun, K., Garzon, J. L., Cimprich, K. A., & Kay, M. A. (2024) · Molecular Therapy

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 4

AAV-mediated genome editing is influenced by the formation of R-loops

Puzzo, F., Crossley, M. P., Goswami, A., Zhang, F., Pekrun, K., Garzon, J. L., Cimprich, K. A., & Kay, M. A. (2024) · Molecular Therapy

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 4

253. Expanded Packaging Capacity of AAV by Lumenal Charge Alteration

Tiffany, M., & Kay, M. A. (2016) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 4