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

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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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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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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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Longitudinal Deformation of the Right Ventricle in Hypoplastic Left Heart Syndrome: A Comparative Study of 2D-Feature Tracking Magnetic Resonance Imaging and 2D-Speckle Tracking Ec... von Salehi Ravesh, Mona, Rickers, Carsten, Bannert, Finn Jonathan, Hautemann, David, Al Bulushi, Abdullah, Gabbert, Dominik Daniel, Wegner, Philip, Kis, Eva, Hansen, Jan Hinnerk, Jerosch-Herold, Michaeal, Kramer, H.-H., Logoteta, Jana

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