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Articles by Jonathan Ark
In vivo site-specific engineering to reprogram T cells
Abstract Engineered T cells, reprogrammed to express chimeric antigen receptors (CAR) or T cell receptors (TCR), have transformed cancer treatment and are being explored as therapeutics for autoimmune and infectious diseases. Enhancing T cell function through genome editing, either by disrupting endogenous genes or precisely inserting DNA payloads, has shown considerable promise1. However, the ex vivo manufacturing process is lengthy and costly, limiting accessibility of these therapies.
In vivo engineering of murine T cells using the evolved adeno-associated virus variant Ark313
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Keywords T cell engineering in vivo gene editing in vivo delivery AAV Ark313 mouse T cells immune therapy knockin gene targeting Introduction Mouse models are essential for building our understanding of T cell biology and for the development of immunotherapies.
By William A Nyberg, Charlotte Wang, Jonathan Ark, Chang Liu, Sylvanie Clouden, Anita Qualls, Sofia E. Caryotakis, Elina Wells, Katherine Simon, Celeste Garza, Pierre-Louis Bernard, Maya Lopez-Ichikawa, Zhongmei Li, Jin Ah Seo, Gabriella R. Kimmerly, Joseph J. Muldoon, Peixin Chen, Mingcheng Li, Hong-Erh Liang, Kelly Kersten, Alan Rosales, Nicholas F. Kuhn, Chun Jimmie Ye, James Gardner, Ari B. Molofsky, Roberto R. Ricardo-Gonzalez, Aravind Asokan, Justin Eyquem
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Cell Press
Verified
A new era in medicine: unraveling the evolution and future of gene therapy
Cell & Gene Therapy Insights 2023; 9(9), 1211–1219 DOI: 10.18609/cgti.2023.159 Over the last decade, gene therapy has rapidly evolved and has led to seven gene therapy approvals since 2017 by the US FDA. These gene therapies are potentially cures for previously incurable diseases. The field has gained tremendous interest from various stakeholders, including patients, providers, investors, and pharmaceutical companies.
An evolved AAV variant enables efficient genetic engineering of murine T cells
Ark313 enables efficient gene targeting in primary murine T cells We hypothesized that the superior transduction of primary murine T cells by Ark313 could be applied to gene editing. We first tested the ability of Ark313 to deliver an sgRNA expression cassette. T cells were isolated from Cas9-expressing mice, activated, and transduced with Ark313 expressing either a Trac-targeting sgRNA or a scrambled (SCR) control sgRNA (Figure 3A).
The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction
Virus-Cell Interactions Victoria J. Madigan, Garrett E. Berry, Tyne O. Tyson, Dasean Nardone-White, Jonathan Ark, Zachary C. Elmore, Giridhar Murlidharan, Heather A. Vincent, Aravind Asokan Rozanne M. Sandri-Goldin, Editor DOI: 10.1128/JVI.01604-20 ABSTRACT Adeno-associated viruses (AAVs) are dependoparvoviruses that have proven useful for therapeutic gene transfer; however, our understanding of host factors that influence AAV trafficking and transduction is still evolving.
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