CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy
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
Published in The Journal of clinical investigation (01.04.2010)
Published in The Journal of clinical investigation (01.04.2010)
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Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS
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.
Published in Molecular therapy (03.10.2018)
Published in Molecular therapy (03.10.2018)
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Engineered Capsids for Efficient Gene Delivery to the Retina and Cornea
Frederick, Amy, Sullivan, Jennifer, Liu, Lin, Adamowicz, Matthew, Lukason, Michael, Raymer, Jasmine, Luo, Zhengyu, Jin, Xiaoying, Rao, Kollu Nageswara, O'Riordan, Catherine
Published in Human gene therapy (01.07.2020)
Published in Human gene therapy (01.07.2020)
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Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa
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.
Published in Molecular therapy (05.07.2023)
Published in Molecular therapy (05.07.2023)
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In Vivo Potency Testing of Subretinal rAAV5.hCNGB1 Gene Therapy in the Cngb1 Knockout Mouse Model of Retinitis Pigmentosa
Wagner, Johanna E, Zobel, Lena, Gerhardt, Maximilian J, O'Riordan, Catherine R, Frederick, Amy, Petersen-Jones, Simon M, Biel, Martin, Michalakis, Stylianos
Published in Human gene therapy (01.10.2021)
Published in Human gene therapy (01.10.2021)
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Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors
Burnham, Brenda, Nass, Shelley, Kong, Elton, Mattingly, MaryEllen, Woodcock, Denise, Song, Antonius, Wadsworth, Samuel, Cheng, Seng H, Scaria, Abraham, O'Riordan, Catherine R
Published in Human gene therapy. Part B. Methods (01.12.2015)
Published in Human gene therapy. Part B. Methods (01.12.2015)
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Cardiovascular Disease Risk Factor Profile of Experienced Male Amateur Marathon Runners: A Systematic Review
O’Riordan, Catherine, Savage, Eileen, Newell, Micheál, Flaherty, Gerard, Hartigan MSc, Irene
Published in Sports Health (01.09.2023)
Published in Sports Health (01.09.2023)
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Book Review
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Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy
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
Published in Human gene therapy (01.07.2014)
Published in Human gene therapy (01.07.2014)
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Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis
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.
Published in Molecular therapy. Methods & clinical development (09.03.2023)
Published in Molecular therapy. Methods & clinical development (09.03.2023)
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Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease
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
Published in Molecular therapy. Methods & clinical development (01.01.2016)
Published in Molecular therapy. Methods & clinical development (01.01.2016)
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The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo
Kyostio-Moore, Sirkka, Berthelette, Patricia, Piraino, Susan, Sookdeo, Cathleen, Nambiar, Bindu, Jackson, Robert, Burnham, Brenda, O'Riordan, Catherine R, Cheng, Seng H, Armentano, Donna
Published in Molecular therapy. Methods & clinical development (01.01.2016)
Published in Molecular therapy. Methods & clinical development (01.01.2016)
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Combination Brain and Systemic Injections of AAV Provide Maximal Functional and Survival Benefits in the Niemann-Pick Mouse
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.
Published in Proceedings of the National Academy of Sciences - PNAS (29.05.2007)
Published in Proceedings of the National Academy of Sciences - PNAS (29.05.2007)
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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
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.
Published in Proceedings of the National Academy of Sciences - PNAS (06.12.2005)
Published in Proceedings of the National Academy of Sciences - PNAS (06.12.2005)
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Characteristics of Minimally Oversized Adeno-Associated Virus Vectors Encoding Human Factor VIII Generated Using Producer Cell Lines and Triple Transfection
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
Published in Human gene therapy. Part B. Methods (01.02.2017)
Published in Human gene therapy. Part B. Methods (01.02.2017)
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Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes
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
Published in Molecular therapy. Methods & clinical development (15.06.2018)
Published in Molecular therapy. Methods & clinical development (15.06.2018)
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AAV Vector-Mediated Correction of Brain Pathology in a Mouse Model of Niemann–Pick A Disease
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.
Published in Molecular therapy (01.05.2005)
Published in Molecular therapy (01.05.2005)
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Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain
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
Published in Gene therapy (01.06.2018)
Published in Gene therapy (01.06.2018)
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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 Vectors
Meng, He, Sorrentino, Michelle, Woodcock, Denise, O'Riordan, Catherine, Dhawan, Vijender, Verhagen, Marc, Davies, Claire
Published in Human gene therapy (01.02.2022)
Published in Human gene therapy (01.02.2022)
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