Zobrazeno 1 - 10
of 30
pro vyhledávání: '"Vivian W. Choi"'
Autor:
Nancy Chen, David E. Ehmann, Robert Crooker, Katayoun Derakhchan, Xiaodong Fang, Brian Felice, Elizabeth J. Galbreath, Charles Glaus, Hongbo Gu, Yan Huang, Christine Li, Xing Li, Nan Liu, Kathleen Palmieri, Damir Simic, Joseph Sypek, Susan Thompson, Christopher T. Winkelmann, Vivian W. Choi
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 29, Iss , Pp 286-302 (2023)
Mucopolysaccharidosis II (MPS II) is a rare lysosomal storage disease characterized by deficient activity of iduronate-2-sulfatase (I2S), leading to pathological accumulation of glycosaminoglycans (GAGs) in tissues. We used iduronate-2-sulfatase knoc
Externí odkaz:
https://doaj.org/article/b33a6cf79e06450e92ea9144dc1312b7
Autor:
Nicolina Cristina Sorrentino, Vincenzo Cacace, Maria De Risi, Veronica Maffia, Sandra Strollo, Novella Tedesco, Edoardo Nusco, Noemi Romagnoli, Domenico Ventrella, Yan Huang, Nan Liu, Susan L. Kalled, Vivian W. Choi, Elvira De Leonibus, Alessandro Fraldi
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 333-342 (2019)
Mucopolysaccharidosis type IIIA (MPS-IIIA) is a lysosomal storage disorder (LSD) caused by inherited defect of sulfamidase, a lysosomal sulfatase. MPS-IIIA is one of the most common and severe forms of LSDs with CNS involvement. Presently there is no
Externí odkaz:
https://doaj.org/article/2d49ccd1afa048a9bc751084b264762d
Autor:
Timothy K. MacLachlan, Mark N. Milton, Oliver Turner, Francis Tukov, Vivian W. Choi, Jan Penraat, Marie-Hélène Delmotte, Lydia Michaut, Bruce D. Jaffee, Chad E. Bigelow
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 8, Iss C, Pp 105-120 (2018)
Retinitis pigmentosa is a form of retinal degeneration usually caused by genetic mutations affecting key functional proteins. We have previously demonstrated efficacy in a mouse model of RLBP1 deficiency with a self-complementary AAV8 vector carrying
Externí odkaz:
https://doaj.org/article/7326adf338ee446f8ac97a123971d3ee
Autor:
Timothy K, MacLachlan, Mark N, Milton, Oliver, Turner, Francis, Tukov, Vivian W, Choi, Jan, Penraat, Marie-Hélène, Delmotte, Lydia, Michaut, Bruce D, Jaffee, Chad E, Bigelow
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Retinitis pigmentosa is a form of retinal degeneration usually caused by genetic mutations affecting key functional proteins. We have previously demonstrated efficacy in a mouse model of RLBP1 deficiency with a self-complementary AAV8 vector carrying
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::da6fe270c5cee4cb3dfc1c87ff16704d
https://pubmed.ncbi.nlm.nih.gov/29359172
https://pubmed.ncbi.nlm.nih.gov/29359172
Autor:
Vivian W Choi, Chad E Bigelow, Terri L McGee, Akshata N Gujar, Hui Li, Shawn M Hanks, Joanna Vrouvlianis, Michael Maker, Barrett Leehy, Yiqin Zhang, Jorge Aranda, George Bounoutas, John T Demirs, Junzheng Yang, Richard Ornberg, Yu Wang, Wendy Martin, Kelly R Stout, Gregory Argentieri, Paul Grosenstein, Danielle Diaz, Oliver Turner, Bruce D Jaffee, Seshidhar R Police, Thaddeus P Dryja
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 2, Iss, Pp-(2015)
Molecular Therapy: Methods & Clinical Development, Vol 2, Iss, Pp-(2015)
Recessive mutations in RLBP1 cause a form of retinitis pigmentosa in which the retina, before its degeneration leads to blindness, abnormally slowly recovers sensitivity after exposure to light. To develop a potential gene therapy for this condition,
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::eff765204dc75ac870b4b8722106520c
Publikováno v:
Journal of Virology. 80:10346-10356
Recentstudies have shown that wild-type and recombinant adeno-associated virus (AAV and rAAV) genomes persist in human tissue predominantly as double-stranded (ds) circular episomes derived from input linear single-stranded virion DNA. Using self-com
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::19794b863ea193a97143aed998858fb2
https://doi.org/10.1128/jvi.00841-06
https://doi.org/10.1128/jvi.00841-06
Autor:
Sarah S. Chisolm, Guorong Li, Teresa Borrás, Jeffrey S. Bartlett, W. Xue, Richard Jude Samulski, Vivian W. Choi
Publikováno v:
The Journal of Gene Medicine. 8:589-602
Background Glaucoma is a chronic eye disease which leads to irreversible blindness. The trabecular meshwork tissue controls intraocular pressure (IOP), which is the major risk factor for glaucoma. Gene therapy treatment of chronic diseases requires t
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::983c49f2ea073011d1f28e82274cf3c1
https://doi.org/10.1002/jgm.886
https://doi.org/10.1002/jgm.886
Publikováno v:
Journal of Virology. 79:6801-6807
Hairpin DNA ends are evolutionarily conserved intermediates in DNA recombination. The hairpin structures present on the ends of the adeno-associated virus (AAV) genome are substrates for recombination that give rise to persistent circular and concate
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::dc01a4ef007750f8b14fc06d288b930d
https://doi.org/10.1128/jvi.79.11.6801-6807.2005
https://doi.org/10.1128/jvi.79.11.6801-6807.2005
Publikováno v:
Current Gene Therapy. 5:299-310
In recent years, significant efforts have been made on studying and engineering adeno-associated virus (AAV) capsid, in order to increase efficiency in targeting specific cell types that are non-permissive to wild type (wt) viruses and to improve eff
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b335f96fdb2f163b6d0fe7a3ecb2d5f0
https://doi.org/10.2174/1566523054064968
https://doi.org/10.2174/1566523054064968
Autor:
Vivian W. Choi, Robin M. Meyers, Amelia N. Chang, Frederick W. Alt, Ali A. Zarrin, Roberto Chiarle, Bjoern Schwer, Gregory T. Marecki, Junchao Dong, Monica Gostissa
Publikováno v:
Proceedings of the National Academy of Sciences
Antibody class switch recombination (CSR) in B lymphocytes joins two DNA double-strand breaks (DSBs) lying 100-200 kb apart within switch (S) regions in the immunoglobulin heavy-chain locus (IgH). CSR-activated B lymphocytes generate multiple S-regio
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::369ce66ba42a300294ecc08c9a76e516