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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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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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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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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Competing barnacle species with a time dependent reproduction rate
Gallagher, M.C., Arnold, M., Kadaub, E., Culloty, S., O’Riordan, R.M., McAllen, R., Rachinskii, D.
Published in Theoretical population biology (01.02.2020)
Published in Theoretical population biology (01.02.2020)
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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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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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Impacts of microcystins on the feeding behaviour and energy balance of zebra mussels, Dreissena polymorpha: A bioenergetics approach
Juhel, G., Davenport, J., O’Halloran, J., Culloty, S.C., O’Riordan, R.M., James, K.F., Furey, A., Allis, O.
Published in Aquatic toxicology (12.10.2006)
Published in Aquatic toxicology (12.10.2006)
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Temporal variation of Meiogymnophallus minutus infections in the first and second intermediate host
Fermer, J., Culloty, S.C., Kelly, T.C., O'Riordan, R.M.
Published in Journal of helminthology (01.12.2010)
Published in Journal of helminthology (01.12.2010)
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Spatial variation in the recruitment of the intertidal barnacles Chthamalus montagui Southward and Chthamalus stellatus (Poli) (Crustacea: Cirripedia) over an European scale
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Published in Journal of experimental marine biology and ecology (30.06.2004)
Published in Journal of experimental marine biology and ecology (30.06.2004)
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Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis
Cheng, Seng H., Gregory, Richard J., Marshall, John, Paul, Sucharita, Souza, David W., White, Gary A., O'Riordan, Catherine R., Smith, Alan E.
Published in Cell (16.11.1990)
Published in Cell (16.11.1990)
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Infection of Mytilus edulis by the trematode Echinostephilla patellae (Digenea: Philophthalmidae)
Prinz, K., Kelly, T.C., O'Riordan, R.M., Culloty, S.C.
Published in Journal of helminthology (01.06.2010)
Published in Journal of helminthology (01.06.2010)
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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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