CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10 von Ruan, Guo-Xiang, Barry, Elizabeth, Yu, Dan, Lukason, Michael, Cheng, Seng H., Scaria, Abraham

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Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis von Kordasiewicz, Holly B., Stanek, Lisa M., Wancewicz, Edward V., Mazur, Curt, McAlonis, Melissa M., Pytel, Kimberly A., Artates, Jonathan W., Weiss, Andreas, Cheng, Seng H., Shihabuddin, Lamya S., Hung, Gene, Bennett, C. Frank, Cleveland, Don W.

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Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial von Heier, Jeffrey S, MD, Kherani, Saleema, MD, Desai, Shilpa, MD, Dugel, Pravin, MD, Kaushal, Shalesh, MD, Cheng, Seng H, PhD, Delacono, Cheryl, OD, Purvis, Annie, MSPH, Richards, Susan, PhD, Le-Halpere, Annaig, PharmD, Connelly, John, MBA, Wadsworth, Samuel C, PhD, Varona, Rafael, MD, Buggage, Ronald, MD, Scaria, Abraham, PhD, Campochiaro, Peter A, Prof

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Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models von Sardi, S. Pablo, Viel, Catherine, Clarke, Jennifer, Treleaven, Christopher M., Richards, Amy M., Park, Hyejung, Olszewski, Maureen A., Dodge, James C., Marshall, John, Makino, Elina, Wang, Bing, Sidman, Richard L., Cheng, Seng H., Shihabuddin, Lamya S.

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy von Wheeler, Thurman M., Leger, Andrew J., Pandey, Sanjay K., MacLeod, A. Robert, Nakamori, Masayuki, Cheng, Seng H., Wentworth, Bruce M., Bennett, C. Frank, Thornton, Charles A.

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Gene therapy for the neurological manifestations in lysosomal storage disorders von Cheng, Seng H.

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Fetal gene therapy for neurodegenerative disease of infants von Massaro, Giulia, Mattar, Citra N. Z., Wong, Andrew M. S., Sirka, Ernestas, Buckley, Suzanne M. K., Herbert, Bronwen R., Karlsson, Stefan, Perocheau, Dany P., Burke, Derek, Heales, Simon, Richard-Londt, Angela, Brandner, Sebastian, Huebecker, Mylene, Priestman, David A., Platt, Frances M., Mills, Kevin, Biswas, Arijit, Cooper, Jonathan D., Chan, Jerry K. Y., Cheng, Seng H., Waddington, Simon N., Rahim, Ahad A.

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Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies von Sardi, S. Pablo, Clarke, Jennifer, Viel, Catherine, Chan, Monyrath, Tamsett, Thomas J., Treleaven, Christopher M., Bu, Jie, Sweet, Lindsay, Passini, Marco A., Dodge, James C., Yu, W. Haung, Sidman, Richard L., Cheng, Seng H., Shihabuddin, Lamya S.

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Glycosphingolipids are modulators of disease pathogenesis in amyotrophic lateral sclerosis von Dodge, James C, Christopher M. Treleaven, Joshua Pacheco, Samantha Cooper, Channa Bao, Marissa Abraham, Mandy Cromwell, S. Pablo Sardi, Wei-Lien Chuang, Richard L. Sidman, Seng H. Cheng, Lamya S. Shihabuddin

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Substrate reduction therapy using Genz‐667161 reduces levels of pathogenic components in a mouse model of neuronopathic forms of Gaucher disease von Blumenreich, Shani, Yaacobi, Chen, Vardi, Ayelet, Barav, Or B., Vitner, Einat B., Park, Hyejung, Wang, Bing, Cheng, Seng H., Sardi, Sergio P., Futerman, Anthony H.

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CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease von Marshall, John, Sun, Ying, Bangari, Dinesh S, Budman, Eva, Park, Hyejung, Nietupski, Jennifer B, Allaire, Amy, Cromwell, Mary A, Wang, Bing, Grabowski, Gregory A, Leonard, John P, Cheng, Seng H

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Gaucher-related synucleinopathies: the examination of sporadic neurodegeneration from a rare (disease) angle von Sardi, S Pablo, Cheng, Seng H, Shihabuddin, Lamya S

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CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy von Sardi, S. Pablo, Clarke, Jennifer, Kinnecom, Cathrine, Tamsett, Thomas J., Li, Lingyun, Stanek, Lisa M., Passini, Marco A., Grabowski, Gregory A., Schlossmacher, Michael G., Sidman, Richard L., Cheng, Seng H., Shihabuddin, Lamya S.

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Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease von Cabrera-Salazar, Mario A, Deriso, Matthew, Bercury, Scott D, Li, Lingyun, Lydon, John T, Weber, William, Pande, Nilesh, Cromwell, Mandy A, Copeland, Diane, Leonard, John, Cheng, Seng H, Scheule, Ronald K

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Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors von Burnham, Brenda, Nass, Shelley, Kong, Elton, Mattingly, MaryEllen, Woodcock, Denise, Song, Antonius, Wadsworth, Samuel, Cheng, Seng H, Scaria, Abraham, O'Riordan, Catherine R

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Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes von Nass, Shelley A, Mattingly, Maryellen A, Woodcock, Denise A, Burnham, Brenda L, Ardinger, Jeffrey A, Osmond, Shayla E, Frederick, Amy M, Scaria, Abraham, Cheng, Seng H, O'Riordan, Catherine R

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Metabolic signatures of amyotrophic lateral sclerosis reveal insights into disease pathogenesis von Dodge, James C., Treleaven, Christopher M., Fidler, Jonathan A., Tamsett, Thomas J., Bao, Channa, Searles, Michelle, Taksir, Tatyana V., Misra, Kuma, Sidman, Richard L., Cheng, Seng H., Shihabuddin, Lamya S.

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Inhibiting Glycosphingolipid Synthesis Improves Glycemic Control and Insulin Sensitivity in Animal Models of Type 2 Diabetes von HONGMEI ZHAO, PRZYBYLSKA, Malgorzata, WU, I-Huan, JINHUA ZHANG, SIEGEL, Craig, KOMARNITSKY, Svetlana, YEW, Nelson S, CHENG, Seng H

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Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease von Marshall, John, Ashe, Karen M, Bangari, Dinesh, McEachern, KerryAnne, Chuang, Wei-Lien, Pacheco, Joshua, Copeland, Diane P, Desnick, Robert J, Shayman, James A, Scheule, Ronald K, Cheng, Seng H

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Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy von Passini, Marco A, Bu, Jie, Richards, Amy M, Treleaven, Christopher M, Sullivan, Jennifer A, O'Riordan, Catherine R, Scaria, Abraham, Kells, Adrian P, Samaranch, Lluis, San Sebastian, Waldy, Federici, Thais, Fiandaca, Massimo S, Boulis, Nicholas M, Bankiewicz, Krystof S, Shihabuddin, Lamya S, Cheng, Seng H

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