Entering the Modern Era of Gene Therapy von Anguela, Xavier M, High, Katherine A

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Immune responses to AAV vectors: overcoming barriers to successful gene therapy von Mingozzi, Federico, High, Katherine A.

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Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges von Mingozzi, Federico, High, Katherine A.

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Turning genes into medicines—what have we learned from gene therapy drug development in the past decade? von High, Katherine A.

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Gene therapy comes of age von Dunbar, Cynthia E, High, Katherine A, Joung, J Keith, Kohn, Donald B, Ozawa, Keiya, Sadelain, Michel

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Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape von Mingozzi, Federico, High, Katherine A

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Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B von George, Lindsey A., Ragni, Margaret V., Rasko, John E.J., Raffini, Leslie J., Samelson-Jones, Benjamin J., Ozelo, Margareth, Hazbon, Maria, Runowski, Alexa R., Wellman, Jennifer A., Wachtel, Katie, Chen, Yifeng, Anguela, Xavier M., Kuranda, Klaudia, Mingozzi, Federico, High, Katherine A.

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In vivo genome editing of the albumin locus as a platform for protein replacement therapy von Sharma, Rajiv, Anguela, Xavier M., Doyon, Yannick, Wechsler, Thomas, DeKelver, Russell C., Sproul, Scott, Paschon, David E., Miller, Jeffrey C., Davidson, Robert J., Shivak, David, Zhou, Shangzhen, Rieders, Julianne, Gregory, Philip D., Holmes, Michael C., Rebar, Edward J., High, Katherine A.

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Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 t... von Bennett, Jean, Prof, Wellman, Jennifer, MSc, Marshall, Kathleen A, COT, McCague, Sarah, BA, Ashtari, Manzar, PhD, DiStefano-Pappas, Julie, BA, Elci, Okan U, PhD, Chung, Daniel C, DO, Sun, Junwei, MSc, Wright, J Fraser, PhD, Cross, Dominique R, MPH, Aravand, Puya, BA, Cyckowski, Laura L, BA, Bennicelli, Jeannette L, PhD, Mingozzi, Federico, PhD, Auricchio, Alberto, MD, Pierce, Eric A, MD, Ruggiero, Jason, MD, Leroy, Bart P, Prof, Simonelli, Francesca, Prof, High, Katherine A, MD, Maguire, Albert M, Prof

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Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer von Buchlis, George, Podsakoff, Gregory M., Radu, Antonetta, Hawk, Sarah M., Flake, Alan W., Mingozzi, Federico, High, Katherine A.

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Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2 von Testa, Francesco, MD, PhD, Maguire, Albert M., MD, Rossi, Settimio, MD, Pierce, Eric A., MD, PhD, Melillo, Paolo, PhD, Marshall, Kathleen, COT, Banfi, Sandro, MD, Surace, Enrico M., DVM, Sun, Junwei, MS, Acerra, Carmela, BA, Wright, J. Fraser, PhD, Wellman, Jennifer, MS, High, Katherine A., MD, Auricchio, Alberto, MD, Bennett, Jean, MD, PhD, Simonelli, Francesca, MD

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Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis von Maguire, Maureen G, Maguire, Albert M, Simonelli, Francesca, Pierce, Eric A, Pugh, Edward N, Mingozzi, Federico, Bennicelli, Jeannette, Banfi, Sandro, Marshall, Kathleen A, Testa, Francesco, Surace, Enrico M, Rossi, Settimio, Lyubarsky, Arkady, Arruda, Valder R, Konkle, Barbara, Stone, Edwin, Sun, Junwei, Jacobs, Jonathan, Dell'Osso, Lou, Hertle, Richard, Ma, Jian-xing, Redmond, T. Michael, Zhu, Xiaosong, Hauck, Bernd, Zelenaia, Olga, Shindler, Kenneth S, Wright, J. Fraser, Volpe, Nicholas J, McDonnell, Jennifer Wellman, Auricchio, Alberto, High, Katherine A, Bennett, Jean

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Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant von McIntosh, Jenny, Lenting, Peter J., Rosales, Cecilia, Lee, Doyoung, Rabbanian, Samira, Raj, Deepak, Patel, Nishil, Tuddenham, Edward G.D., Christophe, Olivier D., McVey, John H., Waddington, Simon, Nienhuis, Arthur W., Gray, John T., Fagone, Paolo, Mingozzi, Federico, Zhou, Shang-Zhen, High, Katherine A., Cancio, Maria, Ng, Catherine Y.C., Zhou, Junfang, Morton, Christopher L., Davidoff, Andrew M., Nathwani, Amit C.

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Robust ZFN-mediated genome editing in adult hemophilic mice von Anguela, Xavier M., Sharma, Rajiv, Doyon, Yannick, Miller, Jeffrey C., Li, Hojun, Haurigot, Virginia, Rohde, Michelle E., Wong, Sunnie Y., Davidson, Robert J., Zhou, Shangzhen, Gregory, Philip D., Holmes, Michael C., High, Katherine A.

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Gene Therapy von High, Katherine A, Roncarolo, Maria G

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Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial von Maguire, Albert M, MD, High, Katherine A, Prof, Auricchio, Alberto, Prof, Wright, J Fraser, PhD, Pierce, Eric A, MD, Testa, Francesco, MD, Mingozzi, Federico, PhD, Bennicelli, Jeannette L, PhD, Ying, Gui-shuang, PhD, Rossi, Settimio, MD, Fulton, Ann, Prof, Marshall, Kathleen A, COT, Banfi, Sandro, MD, Chung, Daniel C, DO, Morgan, Jessica IW, PhD, Hauck, Bernd, PhD, Zelenaia, Olga, PhD, Zhu, Xiaosong, MD, Raffini, Leslie, MD, Coppieters, Frauke, PhD, De Baere, Elfride, PhD, Shindler, Kenneth S, MD, Volpe, Nicholas J, Prof, Surace, Enrico M, DVM, Acerra, Carmela, BA, Lyubarsky, Arkady, PhD, Redmond, T Michael, PhD, Stone, Edwin, Prof, Sun, Junwei, MBA, McDonnell, Jennifer Wellman, MS, Leroy, Bart P, Prof, Simonelli, Francesca, MD, Bennett, Jean, Prof

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Clinical development of gene therapy: results and lessons from recent successes von Kumar, Sandeep Rp, Markusic, David M, Biswas, Moanaro, High, Katherine A, Herzog, Roland W

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Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid von Vercauteren, Koen, Hoffman, Brad E, Zolotukhin, Irene, Keeler, Geoffrey D, Xiao, Jing W, Basner-Tschakarjan, Etiena, High, Katherine A, Ertl, Hildegund CJ, Rice, Charles M, Srivastava, Arun, de Jong, Ype P, Herzog, Roland W

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Assessing the potential for AAV vector genotoxicity in a murine model von Li, Hojun, Malani, Nirav, Hamilton, Shari R., Schlachterman, Alexander, Bussadori, Giulio, Edmonson, Shyrie E., Shah, Rachel, Arruda, Valder R., Mingozzi, Federico, Fraser Wright, J., Bushman, Frederic D., High, Katherine A.

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Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies von Markusic, David M., Hoffman, Brad E., Perrin, George Q., Nayak, Sushrusha, Wang, Xiaomei, LoDuca, Paul A., High, Katherine A., Herzog, Roland W.

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