Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs

by Callan, Mary Beth, Haskins, Mark E., Wang, Ping, Zhou, Shangzhen, High, Katherine A., Arruda, Valder R.
Published 2016

Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B

by Nayak, Sushrusha, Sarkar, Debalina, Perrin, George Q., Moghimi, Babak, Hoffman, Brad E., Zhou, Shangzhen, Byrne, Barry J., Herzog, Roland W.
Published 2011

Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery

by Brown, Harrison C, Wright, J Fraser, Zhou, Shangzhen, Lytle, Allison M, Shields, Jordan E, Spencer, H Trent, Doering, Christopher B
Published 2014

Axonal transport of adeno-associated viral vectors is serotype-dependent

by Salegio, Ernesto A., Samaranch, Lluis, Kells, Adrian P., Mittermeyer, Gabriele, San Sebastian, Waldy, Zhou, Shangzhen, Beyer, Janine, Forsayeth, John, Bankiewicz, Krystof S.
Published 2012

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B

by Mingozzi, Federico, Chen, Yifeng, Murphy, Samuel L, Edmonson, Shyrie C, Tai, Alex, Price, Sandra D, Metzger, Mark E, Zhou, Shangzhen, Wright, J Fraser, Donahue, Robert E, Dunbar, Cynthia E, High, Katherine A
Published 2012

AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models

by Vasireddy, Vidyullatha, Mills, Jason A., Gaddameedi, Rajashekhar, Basner-Tschakarjan, Etiena, Kohnke, Monika, Black, Aaron D., Alexandrov, Krill, Zhou, Shangzhen, Maguire, Albert M., Chung, Daniel C., Mac, Helen, Sullivan, Lisa, Gadue, Paul, Bennicelli, Jeannette L., French, Deborah L., Bennett, Jean
Published 2013

Treatment of Diabetes and Long-Term Survival After Insulin and Glucokinase Gene Therapy

by Callejas, David, Mann, Christopher J., Ayuso, Eduard, Lage, Ricardo, Grifoll, Iris, Roca, Carles, Andaluz, Anna, Ruiz-de Gopegui, Rafael, Montané, Joel, Muñoz, Sergio, Ferre, Tura, Haurigot, Virginia, Zhou, Shangzhen, Ruberte, Jesús, Mingozzi, Federico, High, Katherine A., Garcia, Felix, Bosch, Fatima
Published 2013

Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes

by 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
Published 2013

Correction: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models

by Vasireddy, Vidyullatha, Mills, Jason A., Gaddameedi, Rajashekhar, Basner-Tschakarjan, Etiena, Kohnke, Monika, Black, Aaron D., Alexandrov, Krill, Zhou, Shangzhen, Maguire, Albert M., Chung, Daniel C., Mac, Helen, Sullivan, Lisa, Gadue, Paul, Bennicelli, Jeannette L., French, Deborah L., Bennett, Jean
Published 2015

In vivo genome editing restores hemostasis in a mouse model of hemophilia

by 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.
Published 2011

Safe, Long-term Hepatic Expression of Anti-HCV shRNA in a Nonhuman Primate Model

by Suhy, David A, Kao, Shih-Chu, Mao, Tin, Whiteley, Laurence, Denise, Hubert, Souberbielle, Bernard, Burdick, Andrew D, Hayes, Kyle, Wright, J Fraser, Lavender, Helen, Roelvink, Peter, Kolykhalov, Alexander, Brady, Kevin, Moschos, Sterghios A, Hauck, Bernd, Zelenaia, Olga, Zhou, Shangzhen, Scribner, Curt, High, Katherine A, Renison, Sara H, Corbau, Romu
Published 2012