Zobrazeno 1 - 10
of 43
pro vyhledávání: '"Catherine R O'Riordan"'
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
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.
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::477ffbc006eb0240ce3eb0682728fa49
https://doi.org/10.1016/j.ymthe.2023.04.005
https://doi.org/10.1016/j.ymthe.2023.04.005
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
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::63ac5d74408f69641bb77f2c7c91f082
https://doi.org/10.1089/hum.2021.123
https://doi.org/10.1089/hum.2021.123
Autor:
Amy Frederick, Stylianos Michalakis, Maximilian Gerhardt, Martin Biel, Lena Zobel, Simon M. Petersen-Jones, Johanna Wagner, Catherine R. O'Riordan
Publikováno v:
Hum Gene Ther
Retinitis pigmentosa type 45 (RP45) is an autosomal-recessively inherited blinding disease caused by mutations in the cyclic nucleotide-gated channel subunit beta 1 (CNGB1) gene. In this study, we developed and tested a novel gene supplementation the
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::290e1dd9d81615ee55f493376c9f4b36
https://europepmc.org/articles/PMC8819509/
https://europepmc.org/articles/PMC8819509/
Publikováno v:
Cell and Gene Therapy Insights. 5:1461-1471
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::afab3df47789f8a7eb81149c646ba1e3
https://doi.org/10.18609/cgti.2019.154
https://doi.org/10.18609/cgti.2019.154
Autor:
Lin Liu, Xiaoying Jin, Matthew Adamowicz, Jasmine Raymer, Michael Lukason, Jennifer Sullivan, Catherine R. O'Riordan, Amy Frederick, Zhengyu Luo, Kollu Nageswara Rao
Publikováno v:
Human gene therapy. 31(13-14)
Adeno-associated viral (AAV) vectors represent an ideal vehicle for human gene transfer. One advantage to the AAV vector system is the availability of multiple naturally occurring serotypes that provide selective tropisms for various target cells. St
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b6088f497117e8641424c149b09ef954
https://pubmed.ncbi.nlm.nih.gov/32578442
https://pubmed.ncbi.nlm.nih.gov/32578442
Autor:
Kousaku Ohno, John Forsayeth, Amir Mahmoodi, Waldy San Sebastian, Amin Mahmoodi, Krystof S. Bankiewicz, Lamya S. Shihabuddin, Savanah Trewman, Jerusha Naidoo, Catherine R. O'Riordan, Piotr Hadaczek, Lisa M. Stanek, John Bringas, Jennifer Sullivan, Christopher Snieckus, Lluis Samaranch
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy, vol 26, iss 10
The present study was designed to characterize transduction of non-human primate brain and spinal cord with a modified adeno-associated virus serotype 2, incapable of binding to theheparan sulfate proteoglycan receptor, referred to as AAV2-HBKO. AAV2
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::43439e3c94e50be5bc9e21e6cd4f7a73
https://doi.org/10.1016/j.ymthe.2018.07.008
https://doi.org/10.1016/j.ymthe.2018.07.008
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
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bbd22f17ee71b12e8454dda2fb9b4c48
http://www.sciencedirect.com/science/article/pii/S2329050117301298
http://www.sciencedirect.com/science/article/pii/S2329050117301298
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
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cf002dec946618254bc797779f1b04a3
https://doi.org/10.1089/hgtb.2015.048
https://doi.org/10.1089/hgtb.2015.048