Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in β-thalassemic mice

由 Lisowski, Leszek, Sadelain, Michel
出版 2007

Supplying Clotting Factors From Hematopoietic Stem Cell–derived Erythroid and Megakaryocytic Lineage Cells

由 Sadelain, Michel, Chang, Alex, Lisowski, Leszek
出版 2009

rAAV-Mediated Tumorigenesis: Still Unresolved After an AAV Assault

由 Valdmanis, Paul N, Lisowski, Leszek, Kay, Mark A
出版 2012

Erythroid-specific Human Factor IX Delivery From In Vivo Selected Hematopoietic Stem Cells Following Nonmyeloablative Conditioning in Hemophilia B Mice

由 Chang, Alex H, Stephan, Matthias T, Lisowski, Leszek, Sadelain, Michel
出版 2008

The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA

由 Lisowski, Leszek, Elazar, Menashe, Chu, Kirk, Glenn, Jeffrey S., Kay, Mark A.
出版 2013

Comparison of transduction efficiency among various lentiviruses containing GFP reporter in BM-HSC transplantation

由 Wang, Nan, Rajasekaran, Narendiran, Hou, Tieying, Lisowski, Leszek, Mellins, Elizabeth D.
出版 2013

Second generation codon optimized minicircle (CoMiC) for non-viral reprogramming of human adult fibroblasts

由 Diecke, Sebastian, Lisowski, Leszek, Mordwinkin, Nicholas M., Kooreman, Nigel G., Wu, Joseph C.
發表在 Methods Mol Biol (2014)

AAV Vectors Containing rDNA Homology Display Increased Chromosomal Integration and Transgene Persistence

由 Wang, Zhongya, Lisowski, Leszek, Finegold, Milton J, Nakai, Hiroyuki, Kay, Mark A, Grompe, Markus
出版 2012

Ribosomal DNA Integrating rAAV-rDNA Vectors Allow for Stable Transgene Expression

由 Lisowski, Leszek, Lau, Ashley, Wang, Zhongya, Zhang, Yue, Zhang, Feijie, Grompe, Markus, Kay, Mark A
出版 2012

Targeting Adeno-Associated Virus Vectors for Local Delivery to Fractures and Systemic Delivery to the Skeleton

由 Lee, Lucinda R., Peacock, Lauren, Lisowski, Leszek, Little, David G., Munns, Craig F., Schindeler, Aaron
發表在 Mol Ther Methods Clin Dev (2019)

Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant

由 Melo, Sandra P, Lisowski, Leszek, Bashkirova, Elizaveta, Zhen, Hanson H, Chu, Kirk, Keene, Douglas R, Marinkovich, M Peter, Kay, Mark A, Oro, Anthony E
出版 2014

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

由 Hayakawa, Jun, Ueda, Takahiro, Lisowski, Leszek, Hsieh, Matthew M., Washington, Kareem, Phang, Oswald, Metzger, Mark, Krouse, Allen, Donahue, Robert E., Sadelain, Michel, Tisdale, John F.
出版 2009

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

由 Lisowski, Leszek, Dane, Allison P., Chu, Kirk, Zhang, Yue, Cunningham, Sharon C., Wilson, Elizabeth M., Nygaard, Sean, Grompe, Markus, Alexander, Ian E., Kay, Mark A.
出版 2013

RNAi induced hepatotoxicity results from loss of the first synthesized isoform of miR-122 in mice

由 Valdmanis, Paul N., Gu, Shuo, Chu, Kirk, Jin, Lan, Zhang, Feijie, Munding, Elizabeth M., Zhang, Yue, Huang, Yong, Kutay, Huban, Ghoshal, Kalpana, Lisowski, Leszek, Kay, Mark A.
發表在 Nat Med (2016)

Neurological Disorders Associated with WWOX Germline Mutations—A Comprehensive Overview

由 Banne, Ehud, Abudiab, Baraa, Abu-Swai, Sara, Repudi, Srinivasa Rao, Steinberg, Daniel J., Shatleh, Diala, Alshammery, Sarah, Lisowski, Leszek, Gold, Wendy, Carlen, Peter L., Aqeilan, Rami I.
發表在 Cells (2021)

Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing

由 Nguyen Tran, Minh Thuan, Mohd Khalid, Mohd Khairul Nizam, Wang, Qi, Walker, Jacqueline K. R., Lidgerwood, Grace E., Dilworth, Kimberley L., Lisowski, Leszek, Pébay, Alice, Hewitt, Alex W.
發表在 Nat Commun (2020)

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

由 Moreno, Ana M., Fu, Xin, Zhu, Jie, Katrekar, Dhruva, Shih, Yu-Ru V., Marlett, John, Cabotaje, Jessica, Tat, Jasmine, Naughton, John, Lisowski, Leszek, Varghese, Shyni, Zhang, Kang, Mali, Prashant
發表在 Mol Ther (2018)

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

由 Moreno, Ana M., Fu, Xin, Zhu, Jie, Katrekar, Dhruva, Shih, Yu-Ru V., Marlett, John, Cabotaje, Jessica, Tat, Jasmine, Naughton, John, Lisowski, Leszek, Varghese, Shyni, Zhang, Kang, Mali, Prashant
發表在 Mol Ther (2020)

Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids

由 Cabanes-Creus, Marti, Navarro, Renina Gale, Liao, Sophia H.Y., Baltazar, Grober, Drouyer, Matthieu, Zhu, Erhua, Scott, Suzanne, Luong, Clement, Wilson, Laurence O.W., Alexander, Ian E., Lisowski, Leszek
發表在 Mol Ther Methods Clin Dev (2021)

Genome Editing of Isogenic Human Induced Pluripotent Stem Cells Recapitulates Long QT Phenotype for Drug Testing

由 Wang, Yongming, Liang, Ping, Lan, Feng, Wu, Haodi, Lisowski, Leszek, Gu, Mingxia, Hu, Shijun, Kay, Mark A., Urnov, Fyodor D., Shinnawi, Rami, Gold, Joseph D., Gepstein, Lior, Wu, Joseph C.
出版 2014