The future of recombinant host defense peptides

by Ramon Roca-Pinilla, Leszek Lisowski, Anna Arís, Elena Garcia-Fruitós
Published 2022-12-01

The intersection of vector biology, gene therapy, and hemophilia

by Leszek Lisowski, Janice M. Staber, J. Fraser Wright, Leonard A. Valentino
Published 2021-08-01

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

by Lucinda R. Lee, Lauren Peacock, Leszek Lisowski, David G. Little, Craig F. Munns, Aaron Schindeler
Published 2019-12-01

Neurological Disorders Associated with WWOX Germline Mutations—A Comprehensive Overview

by Ehud Banne, Baraa Abudiab, Sara Abu-Swai, Srinivasa Rao Repudi, Daniel J. Steinberg, Diala Shatleh, Sarah Alshammery, Leszek Lisowski, Wendy Gold, Peter L. Carlen, Rami I. Aqeilan
Published 2021-04-01

Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys

by Julien Baruteau, Sharon C. Cunningham, Berna Seker Yilmaz, Dany P. Perocheau, Simon Eaglestone, Derek Burke, Adrian J. Thrasher, Simon N. Waddington, Leszek Lisowski, Ian E. Alexander, Paul Gissen
Published 2021-12-01

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

by Minh Thuan Nguyen Tran, Mohd Khairul Nizam Mohd Khalid, Qi Wang, Jacqueline K. R. Walker, Grace E. Lidgerwood, Kimberley L. Dilworth, Leszek Lisowski, Alice Pébay, Alex W. Hewitt
Published 2020-09-01

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

by Marti Cabanes-Creus, Renina Gale Navarro, Sophia H.Y. Liao, Grober Baltazar, Matthieu Drouyer, Erhua Zhu, Suzanne Scott, Clement Luong, Laurence O.W. Wilson, Ian E. Alexander, Leszek Lisowski
Published 2021-06-01

Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity

by Ian P. Winters, Shin-Heng Chiou, Nicole K. Paulk, Christopher D. McFarland, Pranav V. Lalgudi, Rosanna K. Ma, Leszek Lisowski, Andrew J. Connolly, Dmitri A. Petrov, Mark A. Kay, Monte M. Winslow
Published 2017-12-01

A hepatic network of dendritic cells mediates CD4 T cell help outside lymphoid organs

by Kieran English, Rain Kwan, Lauren E. Holz, Claire McGuffog, Jelte M. M. Krol, Daryan Kempe, Tsuneyasu Kaisho, William R. Heath, Leszek Lisowski, Maté Biro, Geoffrey W. McCaughan, David G. Bowen, Patrick Bertolino
Published 2024-02-01

Optogenetic restoration of high sensitivity vision with bReaChES, a red-shifted channelrhodopsin

by Lay Khoon Too, Weiyong Shen, Dario A. Protti, Atomu Sawatari, Dylan A. Black, Catherine A. Leamey, Jin Y. Huang, So-Ra Lee, Ashish E. Mathai, Leszek Lisowski, John Y. Lin, Mark C. Gillies, Matthew P. Simunovic
Published 2022-11-01

Long-term ex situ normothermic perfusion of human split livers for more than 1 week

by Ngee-Soon Lau, Mark Ly, Claude Dennis, Andrew Jacques, Marti Cabanes-Creus, Shamus Toomath, Joanna Huang, Nicole Mestrovic, Paul Yousif, Sumon Chanda, Chuanmin Wang, Leszek Lisowski, Ken Liu, James G. Kench, Geoffrey McCaughan, Michael Crawford, Carlo Pulitano
Published 2023-08-01

Bioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle

by Nicole K. Paulk, Katja Pekrun, Gregory W. Charville, Katie Maguire-Nguyen, Michael N. Wosczyna, Jianpeng Xu, Yue Zhang, Leszek Lisowski, Bryan Yoo, Jose G. Vilches-Moure, Gordon K. Lee, Joseph B. Shrager, Thomas A. Rando, Mark A. Kay
Published 2018-09-01

Novel AAV variants with improved tropism for human Schwann cells

by Matthieu Drouyer, Tak-Ho Chu, Elodie Labit, Florencia Haase, Renina Gale Navarro, Deborah Nazareth, Nicole Rosin, Jessica Merjane, Suzanne Scott, Marti Cabanes-Creus, Adrian Westhaus, Erhua Zhu, Rajiv Midha, Ian E. Alexander, Jeff Biernaskie, Samantha L. Ginn, Leszek Lisowski
Published 2024-06-01

Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8

by Marti Cabanes-Creus, Renina Gale Navarro, Erhua Zhu, Grober Baltazar, Sophia H.Y. Liao, Matthieu Drouyer, Anais K. Amaya, Suzanne Scott, Loan Hanh Nguyen, Adrian Westhaus, Matthias Hebben, Laurence O.W. Wilson, Adrian J. Thrasher, Ian E. Alexander, Leszek Lisowski
Published 2022-03-01

Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes

by Samantha L. Ginn, Anais K. Amaya, Sophia H.Y. Liao, Erhua Zhu, Sharon C. Cunningham, Michael Lee, Claus V. Hallwirth, Grant J. Logan, Szun S. Tay, Anthony J. Cesare, Hilda A. Pickett, Markus Grompe, Kimberley Dilworth, Leszek Lisowski, Ian E. Alexander
Published 2020-02-01

Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution

by Marti Cabanes-Creus, Renina Gale Navarro, Sophia H.Y. Liao, Suzanne Scott, Rodrigo Carlessi, Ramon Roca-Pinilla, Maddison Knight, Grober Baltazar, Erhua Zhu, Matthew Jones, Elena Denisenko, Alistair R.R. Forrest, Ian E. Alexander, Janina E.E. Tirnitz-Parker, Leszek Lisowski
Published 2023-03-01

AAV capsid bioengineering in primary human retina models

by Adrian Westhaus, Steven S. Eamegdool, Milan Fernando, Paula Fuller-Carter, Alicia A. Brunet, Annie L. Miller, Rabab Rashwan, Maddison Knight, Maciej Daniszewski, Grace E. Lidgerwood, Alice Pébay, Alex Hewitt, Giorgia Santilli, Adrian J. Thrasher, Livia S. Carvalho, Anai Gonzalez-Cordero, Robyn V. Jamieson, Leszek Lisowski
Published 2023-12-01

Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality

by Marti Cabanes-Creus, Samantha L. Ginn, Anais K. Amaya, Sophia H.Y. Liao, Adrian Westhaus, Claus V. Hallwirth, Patrick Wilmott, Jason Ward, Kimberley L. Dilworth, Giorgia Santilli, Arkadiusz Rybicki, Hiroyuki Nakai, Adrian J. Thrasher, Adrian C. Filip, Ian E. Alexander, Leszek Lisowski
Published 2019-03-01

Proteome-wide systems genetics identifies UFMylation as a regulator of skeletal muscle function

by Jeffrey Molendijk, Ronnie Blazev, Richard J Mills, Yaan-Kit Ng, Kevin I Watt, Daryn Chau, Paul Gregorevic, Peter J Crouch, James BW Hilton, Leszek Lisowski, Peixiang Zhang, Karen Reue, Aldons J Lusis, James E Hudson, David E James, Marcus M Seldin, Benjamin L Parker
Published 2022-12-01

Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2

by Marti Cabanes-Creus, Adrian Westhaus, Renina Gale Navarro, Grober Baltazar, Erhua Zhu, Anais K. Amaya, Sophia H.Y. Liao, Suzanne Scott, Erwan Sallard, Kimberley L. Dilworth, Arkadiusz Rybicki, Matthieu Drouyer, Claus V. Hallwirth, Antonette Bennett, Giorgia Santilli, Adrian J. Thrasher, Mavis Agbandje-McKenna, Ian E. Alexander, Leszek Lisowski
Published 2020-06-01