-
The State of Gene Therapy: An Interview with Jim Wilson
James M. Wilson, Alex Philippidis, and Kevin Davies
-
Adeno-associated virus-mediated trastuzumab delivery to the central nervous system for human epidermal growth factor receptor 2+ brain metastasis
Marcela S Werner, Shweta Aras, Ashleigh R Morgan, Jillian Roamer, Nesteene J Param, Kanyin Olagbegi, R Jason Lamontagne, Jenny A Greig, James M Wilson
-
In situ combinatorial synthesis of degradable branched lipidoids for systemic delivery of mRNA therapeutics and gene editors
Xuexiang Han, Junchao Xu, Ying Xu, Mohamad-Gabriel Alameh, Lulu Xue, Ningqiang Gong, Rakan El-Mayta, Rohan Palanki, Claude C Warzecha, Gan Zhao, Andrew E Vaughan, James M Wilson, Drew Weissman, Michael J Mitchell
-
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury
Juliette Hordeaux, R Jason Lamontagne, Chunjuan Song, George Buchlis, Cecilia Dyer, Elizabeth L Buza, Ali Ramezani, Erik Wielechowski, Jenny A Greig, Jessica A Chichester, Peter Bell, James M Wilson
-
Magnetic Resonance Imaging-Guided Frameless Stereotactic Injections of the Bilateral Cerebellar Dentate Nuclei in Nonhuman Primates: Technical Note
Liming Qiu, Emily Xu, Sydney Chambule, Philip LaTourette II, Cecilia D Dyer, Chelsea K Wallace, Rachel Donocoff, James M Wilson, Timothy H Lucas, H Isaac Chen
-
Integrated vector genomes may contribute to long-term expression in primate liver after AAV administration
Jenny A Greig, Kelly M Martins, Camilo Breton, R Jason Lamontagne, Yanqing Zhu, Zhenning He, John White, Jing-Xu Zhu, Jessica A Chichester, Qi Zheng , Zhe Zhang, Peter Bell, Lili Wang, James M Wilson
-
Prevalent and Disseminated Recombinant and Wild-Type Adeno-Associated Virus Integration in Macaques and Humans
Kelly M Martins, Camilo Breton, Qi Zheng, Zhe Zhang, Caitlin Latshaw, Jenny A Greig, James M Wilson
-
Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy
Teerapat Rojsajjakul, Juliette J Hordeaux, Gourav R Choudhury, Christian J Hinderer, Clementina Mesaros, James M Wilson, Ian A Blair
-
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid
Jonathan B Rosenberg, Edward K Fung, Jonathan P Dyke, Bishnu P De, Howard Lou, James M Kelly, Layla Reejhsinghani, Rodolfo J Ricart Arbona, Dolan Sondhi, Stephen M Kaminsky, Nathalie Cartier, Christian Hinderer, Juliette Hordeaux, James M Wilson, Douglas J Ballon, Ronald G Crystal
-
Adeno-associated virus-vectored erythropoietin gene therapy for anemia in cats with chronic kidney disease
Shelly L Vaden, Allison R Kendall, Jonathan D Foster, Heidi L New, Jane S Eagleson, Jacky L May, Anne M Traas, Matthew J Wilson, Beth H McIntyre, Christian J Hinderer, Lauren K Olenick, James M Wilson
-
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques
Alexander Kistner, Jessica A Chichester, Lili Wang, Roberto Calcedo, Jenny A Greig, Leah N Cardwell, Margaret C Wright, Julien Couthouis, Sunjay Sethi, Brian E McIntosh, Kathleen McKeever, Samuel Wadsworth, James M Wilson, Emil Kakkis, Barbara A Sullivan
-
Significant Differences in Capsid Properties and Potency Between Adeno-Associated Virus Vectors Produced in Sf9 and HEK293 Cells
April Giles, Martin Lock, Shu-Jen Chen, Kevin Turner, Gregg Wesolowski, Andrew Prongay, Boris N. Petkov, Kanyin Olagbegi, Hanying Yan, and James M. Wilson
-
Vector Affinity and Receptor Distribution Define Tissue-Specific Targeting in an Engineered AAV Capsid
R. Alexander Martino, Qiang Wang, Hao Xu, Gui Hu, Peter Bell, Edgardo J. Arroyo, Joshua J. Sims, James M. Wilson
-
Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase
Juliette Hordeaux, Ali Ramezani, Steve Tuske, Nickita Mehta, Chunjuan Song, Anna Lynch, Katherine Lupino, Jessica A. Chichester, Elizabeth L. Buza, Cecilia Dyer, Hongwei Yu, Peter Bell, Jill M. Weimer, Hung Do, James M. Wilson
-
Neonatal Fc Receptor Inhibition Enables Adeno-Associated Virus Gene Therapy Despite Pre-Existing Humoral Immunity
Makoto Horiuchi, Christian J. Hinderer, Hailey N. Shankle, Peter M. Hayashi, Jessica A. Chichester, Casey Kissel, Peter Bell, Cecilia Dyer, James M. Wilson
-
Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model
Jenny A. Greig, Joanna K. Chorazeczewski, Vivek Chowdhary, Melanie K. Smith, Matthew Jennis, James C. Tarrant, Elizabeth L. Buza, Kimberly Coughlan, Paolo G.V. Martini, James M. Wilson