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

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Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema von Qiu, Ting, Chiuchiolo, Maria J., Whaley, Adele S., Russo, Anthony R., Sondhi, Dolan, Kaminsky, Stephen M., Crystal, Ronald G., Pagovich, Odelya E.

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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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Long‐term functional correction of cystathionine β‐synthase deficiency in mice by adeno‐associated viral gene therapy von Lee, Hyung‐Ok, Salami, Christiana O., Sondhi, Dolan, Kaminsky, Stephen M., Crystal, Ronald G., Kruger, Warren D.

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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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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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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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Genetic Modification of the Lung Directed Toward Treatment of Human Disease von Sondhi, Dolan, Stiles, Katie M, De, Bishnu P, Crystal, Ronald G

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Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway von Lojewski, Xenia, Staropoli, John F, Biswas-Legrand, Sunita, Simas, Alexandra M, Haliw, Larissa, Selig, Martin K, Coppel, Scott H, Goss, Kendrick A, Petcherski, Anton, Chandrachud, Uma, Sheridan, Steven D, Lucente, Diane, Sims, Katherine B, Gusella, James F, Sondhi, Dolan, Crystal, Ronald G, Reinhardt, Peter, Sterneckert, Jared, Schöler, Hans, Haggarty, Stephen J, Storch, Alexander, Hermann, Andreas, Cotman, Susan L

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Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA von WORGALL, Stefan, SONDHI, Dolan, CHRISTOS, Paul, MAZUMDAR, Madhu, SOUWEIDANE, Mark M, KAPLITT, Michael G, CRYSTAL, Ronald G, HACKETT, Neil R, KOSOFSKY, Barry, KEKATPURE, Minal V, NEYZI, Nurunisa, DYKE, Jonathan P, BALLON, Douglas, HEIER, Linda, GREENWALD, Bruce M

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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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Untargeted Metabolite Profiling of Cerebrospinal Fluid Uncovers Biomarkers for Severity of Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2, Batten Disease) von Sindelar, Miriam, Dyke, Jonathan P., Deeb, Ruba S., Sondhi, Dolan, Kaminsky, Stephen M., Kosofsky, Barry E., Ballon, Douglas J., Crystal, Ronald G., Gross, Steven S.

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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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Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice von PIGUET, Françoise, SONDHI, Dolan, CARTIER, Nathalie, SEVIN, Caroline, PIRAUD, Monique, FOUQUET, Françoise, HACKETT, Neil R, AHOUANSOU, Ornella, VANIER, Marie-Thérèse, BIECHE, Ivan, AUBOURG, Patrick, CRYSTAL, Ronald G

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Expressed Protein Ligation: A General Method for Protein Engineering von Muir, Tom W., Sondhi, Dolan, Cole, Philip A.

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Enhanced Survival of the LINCL Mouse Following CLN2 Gene Transfer Using the rh.10 Rhesus Macaque-derived Adeno-associated Virus Vector von Sondhi, Dolan, Hackett, Neil R, Peterson, Daniel A, Stratton, Jamie, Baad, Michael, Travis, Kelly M, Wilson, James M, Crystal, Ronald G

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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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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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