Accurate Quantification and Characterization of Adeno-Associated Viral Vectors von Dobnik, David, Kogovšek, Polona, Jakomin, Tjaša, Košir, Nejc, Tušek Žnidarič, Magda, Leskovec, Maja, Kaminsky, Stephen M, Mostrom, Janet, Lee, Hyunmi, Ravnikar, Maja

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Gene therapy for metachromatic leukodystrophy von Rosenberg, Jonathan B., Kaminsky, Stephen M., Aubourg, Patrick, Crystal, Ronald G., Sondhi, Dolan

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Cocaine vaccine dAd5GNE protects against moderate daily and high-dose "binge" cocaine use von Havlicek, David F., Rosenberg, Jonathan B., De, Bishnu P., Hicks, Martin J., Sondhi, Dolan, Kaminsky, Stephen M., Crystal, Ronald G.

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Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy von Rosenberg, Jonathan B., Chen, Alvin, De, Bishnu P., Dyke, Jonathan P., Ballon, Douglas J., Monette, Sebastien, Arbona, Rodolfo J. Ricart, Kaminsky, Stephen M., Crystal, Ronald G., Sondhi, Dolan

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Expression and processing of mature human frataxin after gene therapy in mice von Rojsajjakul, Teerapat, Selvan, Nithya, De, Bishnu, Rosenberg, Jonathan B., Kaminsky, Stephen M., Sondhi, Dolan, Janki, Peter, Crystal, Ronald G., Mesaros, Clementina, Khanna, Richie, Blair, Ian A.

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Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia von Munoz-Zuluaga, Carlos, Gertz, Monica, Yost-Bido, Melissa, Greco, Alessandria, Gorman, Nicholas, Chen, Alvin, Kooner, Vikrum, Rosenberg, Jonathan B, De, Bishnu P, Kaminsky, Stephen M, Borczuk, Alain, Ricart Arbona, Rodolfo J, Martin, Heather R, Monette, Sebastien, Khanna, Richie, Barth, Jay A, Crystal, Ronald G, Sondhi, Dolan

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Anti-hIgE gene therapy of peanut-induced anaphylaxis in a humanized murine model of peanut allergy von Pagovich, Odelya E., MD, Wang, Bo, MD, Chiuchiolo, Maria J., PhD, Kaminsky, Stephen M., PhD, Sondhi, Dolan, PhD, Jose, Clarisse L., BS, Price, Christina C., MD, Brooks, Sarah F., BSc, Mezey, Jason G., PhD, Crystal, Ronald G., MD

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In situ reprogramming to transdifferentiate fibroblasts into cardiomyocytes using adenoviral vectors: Implications for clinical myocardial regeneration von Mathison, Megumi, MD, PhD, Singh, Vivek P., PhD, Chiuchiolo, Maria J., PhD, Sanagasetti, Deepthi, MS, Mao, Yun, MD, Patel, Vivekkumar B., MD, Yang, Jianchang, MD, PhD, Kaminsky, Stephen M., PhD, Crystal, Ronald G., MD, Rosengart, Todd K., MD

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Biology of the Adrenal Gland Cortex Obviates Effective Use of Adeno-Associated Virus Vectors to Treat Hereditary Adrenal Disorders von Markmann, Sandra, De, Bishnu P, Reid, Jasmine, Jose, Clarisse L, Rosenberg, Jonathan B, Leopold, Philip L, Kaminsky, Stephen M, Sondhi, Dolan, Pagovich, Odelya, Crystal, Ronald G

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AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease von Rosenberg, Jonathan B, Kaplitt, Michael G, De, Bishnu P, Chen, Alvin, Flagiello, Thomas, Salami, Christiana, Pey, Eduard, Zhao, Lingzhi, Ricart Arbona, Rodolfo J, Monette, Sebastien, Dyke, Jonathan P, Ballon, Douglas J, Kaminsky, Stephen M, Sondhi, Dolan, Petsko, Gregory A, Paul, Steven M, Crystal, Ronald G

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Evaluation of Compounded Bevacizumab Prepared for Intravitreal Injection von Yannuzzi, Nicolas A, Klufas, Michael A, Quach, Lucy, Beatty, Lauren M, Kaminsky, Stephen M, Crystal, Ronald G, D’Amico, Donald J, Kiss, Szilárd

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“Triplet” polycistronic vectors encoding Gata4, Mef2c, and Tbx5 enhances postinfarct ventricular functional improvement compared with singlet vectors von Mathison, Megumi, MD, PhD, Singh, Vivek P., PhD, Gersch, Robert P., PhD, Ramirez, Maricela O., BS, Cooney, Austin, PhD, Kaminsky, Stephen M., PhD, Chiuchiolo, Maria J., PhD, Nasser, Ahmed, MD, Yang, Jianchang, MD, PhD, Crystal, Ronald G., MD, Rosengart, Todd K., MD

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Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma von Hicks, Martin J, Chiuchiolo, Maria J, Ballon, Douglas, Dyke, Jonathan P, Aronowitz, Eric, Funato, Kosuke, Tabar, Viviane, Havlicek, David, Fan, Fan, Sondhi, Dolan, Kaminsky, Stephen M, Crystal, Ronald G

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Spectrum of ocular manifestations in CLN2-associated batten (Jansky-Bielschowsky) disease correlate with advancing age and deteriorating neurological function von Orlin, Anton, Sondhi, Dolan, Witmer, Matthew T, Wessel, Matthew M, Mezey, Jason G, Kaminsky, Stephen M, Hackett, Neil R, Yohay, Kaleb, Kosofsky, Barry, Souweidane, Mark M, Kaplitt, Michael G, D'Amico, Donald J, Crystal, Ronald G, Kiss, Szilárd

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Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A c... von Rosenberg, Jonathan B, Sondhi, Dolan, Rubin, David G, Monette, Sébastien, Chen, Alvin, Cram, Sara, De, Bishnu P, Kaminsky, Stephen M, Sevin, Caroline, Aubourg, Patrick, Crystal, Ronald G

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Vectored Intracerebral Immunization with the Anti-Tau Monoclonal Antibody PHF1 Markedly Reduces Tau Pathology in Mutant Tau Transgenic Mice von Liu, Wencheng, Zhao, Lingzhi, Blackman, Brittany, Parmar, Mayur, Wong, Man Ying, Woo, Thomas, Yu, Fangmin, Chiuchiolo, Maria J, Sondhi, Dolan, Kaminsky, Stephen M, Crystal, Ronald G, Paul, Steven M

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AAVrh.10 delivery of novel APOE2-Christchurch variant suppresses amyloid and Tau pathology in Alzheimer’s disease mice von Günaydin, Caner, Sondhi, Dolan, Kaminsky, Stephen M., Lephart, Hailey C., Leopold, Philip L., Hackett, Neil R., Khanna, Richie, Crystal, Ronald G.

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Genetic Modification of the AAV5 Capsid with Lysine Residues Results in a Lung-Tropic Liver-Detargeted Gene Transfer Vector von Stiles, Katie M, Frenk, Esther Z, Kaminsky, Stephen M, Crystal, Ronald G

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Assessment of Residual Full-Length SV40 Large T Antigen in Clinical-Grade Adeno-Associated Virus Vectors Produced in 293T Cells von De, Bishnu P, Cram, Sara, Lee, Hyunmi, Rosenberg, Jonathan B, Sondhi, Dolan, Crystal, Ronald G, Kaminsky, Stephen M

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Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models von Zhao, Lingzhi, Gottesdiener, Andrew J., Parmar, Mayur, Li, Mingjie, Kaminsky, Stephen M., Chiuchiolo, Maria J., Sondhi, Dolan, Sullivan, Patrick M., Holtzman, David M., Crystal, Ronald G., Paul, Steven M.

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