Gene therapy comes of age
Dunbar, Cynthia E, High, Katherine A, Joung, J Keith, Kohn, Donald B, Ozawa, Keiya, Sadelain, Michel
Published in Science (American Association for the Advancement of Science) (12.01.2018)
Published in Science (American Association for the Advancement of Science) (12.01.2018)
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Journal Article
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B
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.
Published in Molecular therapy (02.09.2020)
Published in Molecular therapy (02.09.2020)
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Journal Article
In vivo genome editing of the albumin locus as a platform for protein replacement therapy
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.
Published in Blood (08.10.2015)
Published in Blood (08.10.2015)
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Journal Article
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 trial
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
Published in The Lancet (British edition) (13.08.2016)
Published in The Lancet (British edition) (13.08.2016)
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Journal Article
Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2
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
Published in Ophthalmology (Rochester, Minn.) (01.06.2013)
Published in Ophthalmology (Rochester, Minn.) (01.06.2013)
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Journal Article
Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer
Buchlis, George, Podsakoff, Gregory M., Radu, Antonetta, Hawk, Sarah M., Flake, Alan W., Mingozzi, Federico, High, Katherine A.
Published in Blood (29.03.2012)
Published in Blood (29.03.2012)
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Journal Article
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
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
Published in The New England journal of medicine (22.05.2008)
Published in The New England journal of medicine (22.05.2008)
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Journal Article
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
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.
Published in Blood (25.04.2013)
Published in Blood (25.04.2013)
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Journal Article
Overcoming preexisting humoral immunity to AAV using capsid decoys
Mingozzi, Federico, Anguela, Xavier M, Pavani, Giulia, Chen, Yifeng, Davidson, Robert J, Hui, Daniel J, Yazicioglu, Mustafa, Elkouby, Liron, Hinderer, Christian J, Faella, Armida, Howard, Carolann, Tai, Alex, Podsakoff, Gregory M, Zhou, Shangzhen, Basner-Tschakarjan, Etiena, Wright, John Fraser, High, Katherine A
Published in Science translational medicine (17.07.2013)
Published in Science translational medicine (17.07.2013)
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Journal Article
Robust ZFN-mediated genome editing in adult hemophilic mice
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.
Published in Blood (07.11.2013)
Published in Blood (07.11.2013)
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Journal Article
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
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
Published in The Lancet (British edition) (07.11.2009)
Published in The Lancet (British edition) (07.11.2009)
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Journal Article
Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid
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
Published in Molecular therapy (01.06.2016)
Published in Molecular therapy (01.06.2016)
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Journal Article
Assessing the potential for AAV vector genotoxicity in a murine model
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.
Published in Blood (24.03.2011)
Published in Blood (24.03.2011)
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