Zobrazeno 1 - 10
of 51
pro vyhledávání: '"Catherine R O'Riordan"'
Autor:
Shelley A. Nass, Maryellen A. Mattingly, Denise A. Woodcock, Brenda L. Burnham, Jeffrey A. Ardinger, Shayla E. Osmond, Amy M. Frederick, Abraham Scaria, Seng H. Cheng, Catherine R. O’Riordan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 9, Iss , Pp 33-46 (2018)
The generation of clinical good manufacturing practices (GMP)-grade adeno-associated virus (AAV) vectors requires purification strategies that support the generation of vectors of high purity, and that exhibit a good safety and efficacy profile. To d
Externí odkaz:
https://doaj.org/article/c2eeffac7fb04805a98db2eeec81dbf4
Autor:
Laurence M. Occelli, Lena Zobel, Jonathan Stoddard, Johanna Wagner, Nathaniel Pasmanter, Janice Querubin, Lauren M. Renner, Rene Reynaga, Paige A. Winkler, Kelian Sun, Luis Felipe L.P. Marinho, Catherine R. O’Riordan, Amy Frederick, Andreas Lauer, Stephen H. Tsang, William W. Hauswirth, Trevor J. McGill, Martha Neuringer, Stylianos Michalakis, Simon M. Petersen-Jones
Publikováno v:
Molecular Therapy.
Autor:
He Meng, Marc F Verhagen, Denise Woodcock, Claire A. Davies, Catherine R. O'Riordan, Michelle Sorrentino, Vijender Dhawan
Publikováno v:
Human Gene Therapy. 33:202-212
Gene therapy has evolved over the past decade into a promising therapeutic class for treating many intractable diseases. Recombinant adeno-associated virus (AAV) is the most commonly used viral vector for delivering therapeutic genes. Independent of
Autor:
Sirkka Kyostio-Moore, Patricia Berthelette, Susan Piraino, Cathleen Sookdeo, Bindu Nambiar, Robert Jackson, Brenda Burnham, Catherine R O'Riordan, Seng H Cheng, Donna Armentano
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Recombinant adeno-associated viral (rAAV) vectors containing oversized genomes provide transgene expression despite low efficiency packaging of complete genomes. Here, we characterized the properties of oversized rAAV2/8 vectors (up to 5.4 kb) encodi
Externí odkaz:
https://doaj.org/article/dc83fa3e7b174a09885970c39aa9a954
Publikováno v:
Cell and Gene Therapy Insights. 5:1461-1471
Autor:
Maryellen Mattingly, Denise Woodcock, Jeffery Ardinger, Shelley Nass, Seng H. Cheng, Brenda Burnham, Catherine R. O'Riordan, Shayla E. Osmond, Amy Frederick, Abraham Scaria
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 9, Iss, Pp 33-46 (2018)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
The generation of clinical good manufacturing practices (GMP)-grade adeno-associated virus (AAV) vectors requires purification strategies that support the generation of vectors of high purity, and that exhibit a good safety and efficacy profile. To d
Autor:
Brenda Burnham, Abraham Scaria, Samuel C. Wadsworth, Seng H. Cheng, Denise Woodcock, Catherine R. O'Riordan, Elton Kong, Antonius Song, Maryellen Mattingly, Shelley Nass
Publikováno v:
Human Gene Therapy Methods. 26:228-242
Recombinant adeno-associated viral (rAAV) vectors represent a novel class of biopharmaceutical drugs. The production of clinical-grade rAAV vectors for gene therapy would benefit from analytical methods that are able to monitor drug product quality w
Autor:
Lamya S. Shihabuddin, Jie Bu, Seng H. Cheng, Michael Lukason, Abraham Scaria, Jennifer Sullivan, Shayla R Osmond, Catherine R. O'Riordan, Lisa M. Stanek, Elizabeth Barry
Publikováno v:
Gene therapy. 25(3)
The successful application of adeno-associated virus (AAV) gene delivery vectors as a therapeutic paradigm will require efficient gene delivery to the appropriate cells in affected organs. In this study, we utilized a rational design approach to intr
Publikováno v:
Human gene therapy methods. 28(5)
The requirement for robust analytical methods to characterize adeno-associated virus (AAV) vectors is immediate, as the field advances more AAV gene therapies into the clinic and onto commercialization. AAV capsid proteins (VPs) are critical for vira
Autor:
Patricia Berthelette, Bindu Nambiar, Donna Armentano, Cathleen Sookdeo, Brenda Burnham, Susan Piraino, Sirkka Kyostio-Moore, Shelley Nass, Karen A. Vincent, Seng H. Cheng, Catherine R. O'Riordan, David W. Souza, Robert B. Jackson
Publikováno v:
Human gene therapy methods. 28(1)
Several ongoing clinical studies are evaluating recombinant adeno-associated virus (rAAV) vectors as gene delivery vehicles for a variety of diseases. However, the production of vectors with genomes >4.7 kb is challenging, with vector preparations fr