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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Evaluation of inflammation and follicle depletion during ovarian ageing in mice von Lliberos, Carolina, Liew, Seng H., Zareie, Pirooz, La Gruta, Nicole L., Mansell, Ashley, Hutt, Karla

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The capacity of oocytes for DNA repair von Stringer, Jessica M., Winship, Amy, Liew, Seng H., Hutt, Karla

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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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The importance of DNA repair for maintaining oocyte quality in response to anti-cancer treatments, environmental toxins and maternal ageing von Winship, Amy L, Stringer, Jessica M, Liew, Seng H, Hutt, Karla J

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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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Loss of PUMA protects the ovarian reserve during DNA-damaging chemotherapy and preserves fertility von Nguyen, Quynh-Nhu, Zerafa, Nadeen, Liew, Seng H., Morgan, F. Hamish, Strasser, Andreas, Scott, Clare L., Findlay, Jock K., Hickey, Martha, Hutt, Karla J.

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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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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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The Inflammasome Contributes to Depletion of the Ovarian Reserve During Aging in Mice von Lliberos, Carolina, Liew, Seng H., Mansell, Ashley, Hutt, Karla J.

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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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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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Yb-doped WO3 photocatalysts for water oxidation with visible light von Liew, S.L., Zhang, Z., Goh, T.W. Glenn, Subramanian, G.S., Seng, H.L. Debbie, Hor, T.S. Andy, Luo, H.-K., Chi, D.Z.

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Effects of Biocompatible versus Standard Fluid on Peritoneal Dialysis Outcomes von JOHNSON, David W, BROWN, Fiona G, SCHOLLUM, John, SURANYI, Michael, TAN, Seng H, VOSS, David, CLARKE, Margaret, BOUDVILLE, Neil, ELIAS, Tony J, FOO, Marjorie W. Y, JONES, Bernard, KULKARNI, Hemant, LANGHAM, Robyn, RANGANATHAN, Dwarakanathan

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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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