CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy von Passini, Marco A, Bu, Jie, Roskelley, Eric M, Richards, Amy M, Sardi, S Pablo, O'Riordan, Catherine R, Klinger, Katherine W, Shihabuddin, Lamya S, Cheng, Seng H

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Engineered Capsids for Efficient Gene Delivery to the Retina and Cornea von Frederick, Amy, Sullivan, Jennifer, Liu, Lin, Adamowicz, Matthew, Lukason, Michael, Raymer, Jasmine, Luo, Zhengyu, Jin, Xiaoying, Rao, Kollu Nageswara, O'Riordan, Catherine

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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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In Vivo Potency Testing of Subretinal rAAV5.hCNGB1 Gene Therapy in the Cngb1 Knockout Mouse Model of Retinitis Pigmentosa von Wagner, Johanna E., Zobel, Lena, Gerhardt, Maximilian J., O'Riordan, Catherine R., Frederick, Amy, Petersen-Jones, Simon M., Biel, Martin, Michalakis, Stylianos

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Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa von Occelli, Laurence M., Zobel, Lena, Stoddard, Jonathan, Wagner, Johanna, Pasmanter, Nathaniel, Querubin, Janice, Renner, Lauren M., Reynaga, Rene, Winkler, Paige A., Sun, Kelian, Marinho, Luis Felipe L.P., O’Riordan, Catherine R., Frederick, Amy, Lauer, Andreas, Tsang, Stephen H., Hauswirth, William W., McGill, Trevor J., Neuringer, Martha, Michalakis, Stylianos, Petersen-Jones, Simon M.

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Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS von Naidoo, Jerusha, Stanek, Lisa M., Ohno, Kousaku, Trewman, Savanah, Samaranch, Lluis, Hadaczek, Piotr, O’Riordan, Catherine, Sullivan, Jennifer, San Sebastian, Waldy, Bringas, John R., Snieckus, Christopher, Mahmoodi, Amin, Mahmoodi, Amir, Forsayeth, John, Bankiewicz, Krystof S., Shihabuddin, Lamya S.

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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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Physics resources for varied communities von O’Riordan, Catherine

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Cardiovascular Disease Risk Factor Profile of Experienced Male Amateur Marathon Runners: A Systematic Review von O’Riordan, Catherine, Savage, Eileen, Newell, Micheál, Flaherty, Gerard, Hartigan MSc, Irene

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Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis von Boye, Sanford L., O’Riordan, Catherine, Morris, James, Lukason, Michael, Compton, David, Baek, Rena, Elmore, Dana M., Peterson, James.J., Fajardo, Diego, McCullough, K. Tyler, Scaria, Abraham, McVie-Wylie, Alison, Boye, Shannon E.

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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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Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease von Hadaczek, Piotr, Stanek, Lisa, Ciesielska, Agnieszka, Sudhakar, Vivek, Samaranch, Lluis, Pivirotto, Philip, Bringas, John, O'Riordan, Catherine, Mastis, Bryan, San Sebastian, Waldy, Forsayeth, John, Cheng, Seng H, Bankiewicz, Krystof S, Shihabuddin, Lamya S

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The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo von Kyostio-Moore, Sirkka, Berthelette, Patricia, Piraino, Susan, Sookdeo, Cathleen, Nambiar, Bindu, Jackson, Robert, Burnham, Brenda, O'Riordan, Catherine R, Cheng, Seng H, Armentano, Donna

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Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain von Sullivan, Jennifer A., Stanek, Lisa M., Lukason, Michael J., Bu, Jie, Osmond, Shayla R., Barry, Elizabeth A., O’Riordan, Catherine R., Shihabuddin, Lamya S., Cheng, Seng H., Scaria, Abraham

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Gene Transfer of Human Acid Sphingomyelinase Corrects Neuropathology and Motor Deficits in a Mouse Model of Niemann-Pick Type A Disease von James C. Dodge, Jennifer Clarke, Antonius Song, Jie Bu, Yang, Wendy, Tatyana V. Taksir, Denise Griffiths, Michael A. Zhao, Schuchman, Edward H., Cheng, Seng H., Catherine R. O'Riordan, Shihabuddin, Lamya S., Marco A. Passini, Stewart, Gregory R.

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Combination Brain and Systemic Injections of AAV Provide Maximal Functional and Survival Benefits in the Niemann-Pick Mouse von Passini, Marco A., Bu, Jie, Fidler, Jonathan A., Ziegler, Robin J., Foley, Joseph W., Dodge, James C., Yang, Wendy W., Clarke, Jennifer, Taksir, Tatyana V., Griffiths, Denise A., Zhao, Michael A., O'Riordan, Catherine R., Schuchman, Edward H., Shihabuddin, Lamya S., Cheng, Seng H.

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Wanted: future leaders for ESA von O'RIORDAN, CATHERINE, BARON, JILL

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Characteristics of Minimally Oversized Adeno-Associated Virus Vectors Encoding Human Factor VIII Generated Using Producer Cell Lines and Triple Transfection von Nambiar, Bindu, Cornell Sookdeo, Cathleen, Berthelette, Patricia, Jackson, Robert, Piraino, Susan, Burnham, Brenda, Nass, Shelley, Souza, David, O'Riordan, Catherine R, Vincent, Karen A, Cheng, Seng H, Armentano, Donna, Kyostio-Moore, Sirkka

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AAV Vector-Mediated Correction of Brain Pathology in a Mouse Model of Niemann–Pick A Disease von Passini, Marco A., Macauley, Shannon L., Huff, Michael R., Taksir, Tatyana V., Bu, Jie, Wu, I-Huan, Piepenhagen, Peter A., Dodge, James C., Shihabuddin, Lamya S., O'Riordan, Catherine R., Schuchman, Edward H., Stewart, Gregory R.

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Size Exclusion Chromatography with Dual Wavelength Detection as a Sensitive and Accurate Method for Determining the Empty and Full Capsids of Recombinant Adeno-Associated Viral Vec... von Meng, He, Sorrentino, Michelle, Woodcock, Denise, O'Riordan, Catherine, Dhawan, Vijender, Verhagen, Marc, Davies, Claire

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