Lisowski, Leszek

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

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

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

In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation

Moreno, A. M., Fu, X., Zhu, J., Katrekar, D., Shih, Y.-R. V., Marlett, J., Cabotaje, J., Tat, J., Naughton, J., Lisowski, L., et al. (2018) · Molecular Therapy

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 159

High-Throughput In Vitro , Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction

Westhaus, A., Cabanes-Creus, M., Rybicki, A., Baltazar, G., Navarro, R. G., Zhu, E., Drouyer, M., Knight, M., Albu, R. F., Ng, B. H., et al. (2020) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 102

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort

Perocheau, D. P., Cunningham, S. C., Lee, J., Antinao Diaz, J., Waddington, S. N., Gilmour, K., Eaglestone, S., Lisowski, L., Thrasher, A. J., Alexander, I. E., et al. (2019) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 81

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

Characterization of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retina

Kumar, S., Hsiao, Y.-W., Wong, V. H. Y., Aubin, D., Wang, J.-H., Lisowski, L., Rakoczy, E. P., Li, F., Alarcon-Martinez, L., Gonzalez-Cordero, A., et al. (2024) · Proceedings of the National Academy of Sciences

CRISPR and Genetic EngineeringRetinal Development and Disorders

Cited by 19

Transient In Vivo β-Globin Production After Lentiviral Gene Transfer to Hematopoietic Stem Cells in the Nonhuman Primate

Hayakawa, J., Ueda, T., Lisowski, L., Hsieh, M. M., Washington, K., Phang, O., Metzger, M., Krouse, A., Donahue, R. E., Sadelain, M., et al. (2009) · Human Gene Therapy

Virus-based gene therapy researchHematopoietic Stem Cell Transplantation

Cited by 9