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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Agarwood Induction: Current Developments and Future Perspectives von Tan, Cheng Seng, Isa, Nurulhikma Md, Ismail, Ismanizan, Zainal, Zamri

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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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Dimorphic effects of transforming growth factor-β signaling during aortic aneurysm progression in mice suggest a combinatorial therapy for Marfan syndrome von Cook, Jason R, Clayton, Nicholas P, Carta, Luca, Galatioto, Josephine, Chiu, Emily, Smaldone, Silvia, Nelson, Carol A, Cheng, Seng H, Wentworth, Bruce M, Ramirez, Francesco

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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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Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy von Schulz, Martin, Levy, Daniel I., Petropoulos, Christos J., Bashirians, George, Winburn, Ian, Mahn, Matthias, Somanathan, Suryanarayan, Cheng, Seng H., Byrne, Barry J.

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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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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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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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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 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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A Fully Integrated 8-Channel Closed-Loop Neural-Prosthetic CMOS SoC for Real-Time Epileptic Seizure Control von Chen, Wei-Ming, Chiueh, Herming, Chen, Tsan-Jieh, Ho, Chia-Lun, Jeng, Chi, Ker, Ming-Dou, Lin, Chun-Yu, Huang, Ya-Chun, Chou, Chia-Wei, Fan, Tsun-Yuan, Cheng, Ming-Seng, Hsin, Yue-Loong, Liang, Sheng-Fu, Wang, Yu-Lin, Shaw, Fu-Zen, Huang, Yu-Hsing, Yang, Chia-Hsiang, Wu, Chung-Yu

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A 13.56 MHz 40 mW CMOS High-Efficiency Inductive Link Power Supply Utilizing On-Chip Delay-Compensated Voltage Doubler Rectifier and Multiple LDOs for Implantable Medical Devices von Wu, Chung-Yu, Qian, Xin-Hong, Cheng, Ming-Seng, Liang, Yu-An, Chen, Wei-Ming

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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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