A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing von Finn, Jonathan D., Smith, Amy Rhoden, Patel, Mihir C., Shaw, Lucinda, Youniss, Madeleine R., van Heteren, Jane, Dirstine, Tanner, Ciullo, Corey, Lescarbeau, Reynald, Seitzer, Jessica, Shah, Ruchi R., Shah, Aalok, Ling, Dandan, Growe, Jacqueline, Pink, Melissa, Rohde, Ellen, Wood, Kristy M., Salomon, William E., Harrington, William F., Dombrowski, Christian, Strapps, Walter R., Chang, Yong, Morrissey, David V.

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Unlocking the potential of iPSC-derived immune cells: engineering iNK and iT cells for cutting-edge immunotherapy von Fang, Minggang, Allen, Alexander, Luo, Chong, Finn, Jonathan D

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Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy von Finn, Jonathan D., Ozelo, Margareth C., Sabatino, Denise E., Franck, Helen W.G., Merricks, Elizabeth P., Crudele, Julie M., Zhou, Shangzhen, Kazazian, Haig H., Lillicrap, David, Nichols, Timothy C., Arruda, Valder R.

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Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates von Samelson-Jones, Benjamin J., Finn, Jonathan D., Favaro, Patricia, Wright, J. Fraser, Arruda, Valder R.

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Transfection reagent artefact likely accounts for some reports of extracellular vesicle function von McConnell, Russell E., Youniss, Madeleine, Gnanasambandam, Bhargavee, Shah, Palak, Zhang, Wei, Finn, Jonathan D.

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A versatile platform for generating engineered extracellular vesicles with defined therapeutic properties von Dooley, Kevin, McConnell, Russell E., Xu, Ke, Lewis, Nuruddeen D., Haupt, Sonya, Youniss, Madeleine R., Martin, Shelly, Sia, Chang Ling, McCoy, Christine, Moniz, Raymond J., Burenkova, Olga, Sanchez-Salazar, Jorge, Jang, Su Chul, Choi, Bryan, Harrison, Rane A., Houde, Damian, Burzyn, Dalia, Leng, Charan, Kirwin, Katherine, Ross, Nikki L., Finn, Jonathan D., Gaidukov, Leonid, Economides, Kyriakos D., Estes, Scott, Thornton, James E., Kulman, John D., Sathyanarayanan, Sriram, Williams, Douglas E.

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Persistence of Transgene Expression Influences CD8+ T-Cell Expansion and Maintenance following Immunization with Recombinant Adenovirus von FINN, Jonathan D, BASSETT, Jennifer, MILLAR, James B, GRINSHTEIN, Natalie, TENG CHIH YANG, PARSONS, Robin, EVELEGH, Carole, YONGHONG WAN, PARKS, Robin J, BRAMSON, Jonathan L

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Characterization of naturally-occurring humoral immunity to AAV in sheep von Tellez, Joseph, Van Vliet, Kim, Tseng, Yu-Shan, Finn, Jonathan D, Tschernia, Nick, Almeida-Porada, Graça, Arruda, Valder R, Agbandje-McKenna, Mavis, Porada, Christopher D

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AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice von Crudele, Julie M., Finn, Jonathan D., Siner, Joshua I., Martin, Nicholas B., Niemeyer, Glenn P., Zhou, Shangzhen, Mingozzi, Federico, Lothrop, Clinton D., Arruda, Valder R.

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In vivo genome editing restores haemostasis in a mouse model of haemophilia von Li, Hojun, Haurigot, Virginia, Doyon, Yannick, Li, Tianjian, Wong, Sunnie Y., Bhagwat, Anand S., Malani, Nirav, Anguela, Xavier M., Sharma, Rajiv, Ivanciu, Lacramiora, Murphy, Samuel L., Finn, Jonathan D., Khazi, Fayaz R., Zhou, Shangzhen, Paschon, David E., Rebar, Edward J., Bushman, Frederic D., Gregory, Philip D., Holmes, Michael C., High, Katherine A.

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X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua) von Simioni, Paolo, Tormene, Daniela, Tognin, Giulio, Gavasso, Sabrina, Bulato, Cristiana, Iacobelli, Nicholas P, Finn, Jonathan D, Spiezia, Luca, Radu, Claudia, Arruda, Valder R

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Safety of Liver Gene Transfer Following Peripheral Intravascular Delivery of Adeno-Associated Virus (AAV)-5 and AAV-6 in a Large Animal Model von FAVARO, Patricia, FINN, Jonathan D, SINER, Joshua I, FRASER WRIGHT, J, HIGH, Katherine A, ARRUDA, Valder R

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Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells von Martino, Ashley T., Basner-Tschakarjan, Etiena, Markusic, David M., Finn, Jonathan D., Hinderer, Christian, Zhou, Shangzhen, Ostrov, David A., Srivastava, Arun, Ertl, Hildegund C.J., Terhorst, Cox, High, Katherine A., Mingozzi, Federico, Herzog, Roland W.

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The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy von Finn, Jonathan D., Nichols, Timothy C., Svoronos, Nikolaos, Merricks, Elizabeth P., Bellenger, Dwight A., Zhou, Shangshen, Simioni, Paolo, High, Katherine A., Arruda, Valder R.

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Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors von Pien, Gary C, Basner-Tschakarjan, Etiena, Hui, Daniel J, Mentlik, Ashley N, Finn, Jonathan D, Hasbrouck, Nicole C, Zhou, Shangzhen, Murphy, Samuel L, Maus, Marcela V, Mingozzi, Federico, Orange, Jordan S, High, Katherine A

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Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction von Finn, Jonathan D, Hui, Daniel, Downey, Harre D, Dunn, Danielle, Pien, Gary C, Mingozzi, Federico, Zhou, Shangzhen, High, Katherine A

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Strategies to Modulate Immune Responses: A New Frontier for Gene Therapy von Arruda, Valder R, Favaro, Patricia, Finn, Jonathan D

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Empty Capsids and Macrophage Inhibition/Depletion Increase rAAV Transgene Expression in Joints of Both Healthy and Arthritic Mice von Aalbers, Caroline J, Broekstra, Niels, van Geldorp, Mariska, Kramer, Emiel, Ramiro, Sofia, Tak, Paul P, Vervoordeldonk, Margriet J, Finn, Jonathan D

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Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity von Samelson-Jones, Benjamin J, Finn, Jonathan D, George, Lindsey A, Camire, Rodney M, Arruda, Valder R

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Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes von Hui, Daniel J, Basner-Tschakarjan, Etiena, Chen, Yifeng, Davidson, Robert J, Buchlis, George, Yazicioglu, Mustafa, Pien, Gary C, Finn, Jonathan D, Haurigot, Virginia, Tai, Alex, Scott, David W, Cousens, Leslie P, Zhou, Shangzhen, De Groot, Anne S, Mingozzi, Federico

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