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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials
Maguire, Albert M, Russell, Stephen, Wellman, Jennifer A, Chung, Daniel C, Yu, Zi-Fan, Tillman, Amy, Wittes, Janet, Pappas, Julie, Elci, Okan, Marshall, Kathleen A, McCague, Sarah, Reichert, Hannah, Davis, Maria, Simonelli, Francesca, Leroy, Bart P, Wright, J Fraser, High, Katherine A, Bennett, Jean
Published in Ophthalmology (Rochester, Minn.) (01.09.2019)
Published in Ophthalmology (Rochester, Minn.) (01.09.2019)
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Journal Article
Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial
Mishra, Alaknanda, Vijayasarathy, Camasamudram, Cukras, Catherine A., Wiley, Henry E., Sen, H. Nida, Zeng, Yong, Wei, Lisa L., Sieving, Paul A.
Published in Molecular therapy (02.06.2021)
Published in Molecular therapy (02.06.2021)
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Journal Article
AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye
Reichel, Felix F., Dauletbekov, Daniyar L., Klein, Reinhild, Peters, Tobias, Ochakovski, G. Alex, Seitz, Immanuel P., Wilhelm, Barbara, Ueffing, Marius, Biel, Martin, Wissinger, Bernd, Michalakis, Stylianos, Bartz-Schmidt, Karl Ulrich, Fischer, M. Dominik
Published in Molecular therapy (06.12.2017)
Published in Molecular therapy (06.12.2017)
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Journal Article
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes
Mendell, Jerry R., Sahenk, Zarife, Al-Zaidy, Samiah, Rodino-Klapac, Louise R., Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Miller, Natalie, Yalvac, Mehmet, Dvorchik, Igor, Moore-Clingenpeel, Melissa, Flanigan, Kevin M., Church, Kathleen, Shontz, Kim, Curry, Choumpree, Lewis, Sarah, McColly, Markus, Hogan, Mark J., Kaspar, Brian K.
Published in Molecular therapy (05.04.2017)
Published in Molecular therapy (05.04.2017)
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Journal Article
The Assembly-Activating Protein Promotes Stability and Interactions between AAV’s Viral Proteins to Nucleate Capsid Assembly
Maurer, Anna C., Pacouret, Simon, Cepeda Diaz, Ana Karla, Blake, Jessica, Andres-Mateos, Eva, Vandenberghe, Luk H.
Published in Cell reports (Cambridge) (08.05.2018)
Published in Cell reports (Cambridge) (08.05.2018)
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Journal Article
Leber Hereditary Optic Neuropathy Gene Therapy: Adverse Events and Visual Acuity Results of All Patient Groups
Lam, Byron L., Feuer, William J., Davis, Janet L., Porciatti, Vittorio, Yu, Hong, Levy, Robert B., Vanner, Elizabeth, Guy, John
Published in American journal of ophthalmology (01.09.2022)
Published in American journal of ophthalmology (01.09.2022)
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Journal Article
Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression
Mueller, Christian, Chulay, Jeffrey D., Trapnell, Bruce C., Humphries, Margaret, Carey, Brenna, Sandhaus, Robert A., McElvaney, Noel G., Messina, Louis, Tang, Qiushi, Rouhani, Farshid N., Campbell-Thompson, Martha, Fu, Ann Dongtao, Yachnis, Anthony, Knop, David R., Ye, Guo-jie, Brantly, Mark, Calcedo, Roberto, Somanathan, Suryanarayan, Richman, Lee P., Vonderheide, Robert H., Hulme, Maigan A., Brusko, Todd M., Wilson, James M., Flotte, Terence R.
Published in The Journal of clinical investigation (01.12.2013)
Published in The Journal of clinical investigation (01.12.2013)
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Journal Article
AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells
Mingozzi, Federico, Meulenberg, Janneke J., Hui, Daniel J., Basner-Tschakarjan, Etiena, Hasbrouck, Nicole C., Edmonson, Shyrie A., Hutnick, Natalie A., Betts, Michael R., Kastelein, John J., Stroes, Erik S., High, Katherine A.
Published in Blood (03.09.2009)
Published in Blood (03.09.2009)
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Journal Article
Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset
Newman, Nancy J., Yu-Wai-Man, Patrick, Carelli, Valerio, Moster, Mark L., Biousse, Valerie, Vignal-Clermont, Catherine, Sergott, Robert C., Klopstock, Thomas, Sadun, Alfredo A., Barboni, Piero, DeBusk, Adam A., Girmens, Jean François, Rudolph, Günther, Karanjia, Rustum, Taiel, Magali, Blouin, Laure, Smits, Gerard, Katz, Barrett, Sahel, José-Alain, Vignal, Catherine, Hage, Rabih, Catarino, Claudia B., Priglinger, Claudia, Priglinger, Siegfried, Thurau, Stephan, von Livonius, Bettina, Muth, Daniel, Wolf, Armin, Al-Tamami, Jasmina, Pressler, Angelika, Schertler, Cosima, Hildebrandt, Martin, Neuenhahn, Michael, Heilweil, Gad, Tsui, Irena, Hubbard, G. Baker, Hendrick, Andrew, Dattilo, Michael, Peragallo, Jason, Hawy, Eman, DuBois, Med, Lindreth, Gibbs, Deborah, Filho, Alcides Fernandes, Dobbs, Jannah, Carbonelli, Michele, Di Vito, Lidia, Contin, Manuela, Mohamed, Susan, La Morgia, Chiara, Silvestri, Sara, Acheson, James, Eleftheriadou, Maria, Esposti, Simona, Gemenetzi, Maria, Leitch-Devlin, Lauren, Tucker, William R., Jurkute, Neringa, SantaMaria, Melissa, Tollis, Heather, Haller, Julie A., Massini, Maria
Published in Ophthalmology (Rochester, Minn.) (01.05.2021)
Published in Ophthalmology (Rochester, Minn.) (01.05.2021)
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Journal Article
Viral vector platforms within the gene therapy landscape
Bulcha, Jote T., Wang, Yi, Ma, Hong, Tai, Phillip W. L., Gao, Guangping
Published in Signal transduction and targeted therapy (08.02.2021)
Published in Signal transduction and targeted therapy (08.02.2021)
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Journal Article
The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals
Mimuro, Jun, Mizukami, Hiroaki, Shima, Midori, Matsushita, Tadashi, Taki, Masashi, Muto, Shinji, Higasa, Satoshi, Sakai, Michio, Ohmori, Tsukasa, Madoiwa, Seiji, Ozawa, Keiya, Sakata, Yoichi
Published in Journal of medical virology (01.11.2014)
Published in Journal of medical virology (01.11.2014)
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Journal Article
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
Brantly, Mark L, Chulay, Jeffrey D, Wang, Lili, Mueller, Christian, Humphries, Margaret, Spencer, L. Terry, Rouhani, Farshid, Conlon, Thomas J, Calcedo, Roberto, Betts, Michael R, Spencer, Carolyn, Byrne, Barry J, Wilson, James M, Flotte, Terence R
Published in Proceedings of the National Academy of Sciences - PNAS (22.09.2009)
Published in Proceedings of the National Academy of Sciences - PNAS (22.09.2009)
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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
A negative regulator of MAP kinase causes depressive behavior
Duric, Vanja, Banasr, Mounira, Licznerski, Pawel, Schmidt, Heath D, Stockmeier, Craig A, Simen, Arthur A, Newton, Samuel S, Duman, Ronald S
Published in Nature medicine (01.11.2010)
Published in Nature medicine (01.11.2010)
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Journal Article
Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: A double-blind, randomized, controlled trial
Warren Olanow, C., Bartus, Raymond T., Baumann, Tiffany L., Factor, Stewart, Boulis, Nicholas, Stacy, Mark, Turner, Dennis A., Marks, William, Larson, Paul, Starr, Phillip A., Jankovic, Joseph, Simpson, Richard, Watts, Ray, Guthrie, Barton, Poston, Kathleen, Henderson, Jaimie M., Stern, Matthew, Baltuch, Gordon, Goetz, Christopher G., Herzog, Christopher, Kordower, Jeffrey H., Alterman, Ron, Lozano, Andres M., Lang, Anthony E.
Published in Annals of neurology (01.08.2015)
Published in Annals of neurology (01.08.2015)
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Journal Article
Neutralizing antibodies against AAV2, AAV5 and AAV8 in healthy and HIV-1-infected subjects in China: implications for gene therapy using AAV vectors
Liu, Q, Huang, W, Zhang, H, Wang, Y, Zhao, J, Song, A, Xie, H, Zhao, C, Gao, D, Wang, Y
Published in Gene therapy (01.08.2014)
Published in Gene therapy (01.08.2014)
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Journal Article