Zobrazeno 1 - 7
of 7
pro vyhledávání: '"Sebastian Gloskowski"'
Autor:
Christianne E. Strang, Dibyendu Chakraborty, Jim Peterson, Diego Fajardo, Paul D. Gamlin, C. Douglas Witherspoon, Haiyan Jiang, Steven J. Samuelsson, Morgan L. Maeder, Kaitlyn R. Calabro, Shannon E. Boye, Scott Haskett, Sebastian Gloskowski, K. Tyler McCullough, Sanford L. Boye
Publikováno v:
Human Gene Therapy. 30:571-589
Mutations in GUCY2D, the gene encoding retinal guanylate cyclase-1 (retGC1), are the leading cause of autosomal dominant cone-rod dystrophy (CORD6). Significant progress toward clinical application of gene replacement therapy for Leber congenital ama
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::392cdcba89da3c2d5c90495d4de8bcec
https://doi.org/10.1089/hum.2018.193
https://doi.org/10.1089/hum.2018.193
Autor:
Bei Hopkins, Steven J. Samuelsson, Terence Ta, Haiyan Jiang, Dawn Ciulla, Morgan L. Maeder, Joy E. Horng, Ari E. Friedland, Hoson Chao, Timothy Fennell, Georgia Giannoukos, Maxwell N. Skor, Sebastian Gloskowski, Christine D. Wilson, Rina Mepani, Diana Tabbaa, Gregory Gotta, Vic E. Myer, Jennifer A. DaSilva, Reshica Baral, Eugenio Marco, Alexandra Glucksmann, Deepak Reyon, Vidya Dhanapal, David Bumcrot, Luis A. Barrera, Shivangi Joshi, Tongyao Wang, Michael Stefanidakis, Abhishek Dass, Charles F Albright, Clifford Yudkoff, George S. Bounoutas, Scott Haskett, Shen Shen, Pam Stetkiewicz, Hariharan Jayaram
Publikováno v:
Nature Medicine. 25:229-233
Leber congenital amaurosis type 10 is a severe retinal dystrophy caused by mutations in the CEP290 gene1,2. We developed EDIT-101, a candidate genome-editing therapeutic, to remove the aberrant splice donor created by the IVS26 mutation in the CEP290
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::86c0cd9b3d2cffb0a51b7703154f9c31
https://doi.org/10.1038/s41591-018-0327-9
https://doi.org/10.1038/s41591-018-0327-9
Autor:
Nachiket D. Pendse, Basil S. Pawlyk, Zheng-Yi Chen, Sebastian Gloskowski, Qin Liu, Morgan L. Maeder, Veronica Lamas, Eric A. Pierce
Mutations in the USH2A gene are the most common cause of non-syndromic inherited retinal degeneration and Usher syndrome, which is characterized by congenital deafness and progressive vision loss. Development of a vector mediated therapy for USH2A-as
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5f5b0e203955f0fcdb5d92b067234c4f
https://doi.org/10.1101/2020.02.04.934240
https://doi.org/10.1101/2020.02.04.934240
Autor:
K Tyler, McCullough, Sanford L, Boye, Diego, Fajardo, Kaitlyn, Calabro, James J, Peterson, Christianne E, Strang, Dibyendu, Chakraborty, Sebastian, Gloskowski, Scott, Haskett, Steven, Samuelsson, Haiyan, Jiang, C Douglas, Witherspoon, Paul D, Gamlin, Morgan L, Maeder, Shannon E, Boye
Publikováno v:
Hum Gene Ther
Mutations in GUCY2D, the gene encoding retinal guanylate cyclase-1 (retGC1), are the leading cause of autosomal dominant cone–rod dystrophy (CORD6). Significant progress toward clinical application of gene replacement therapy for Leber congenital a
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::1cf316d5aca359e15a75ee8460f80921
https://pubmed.ncbi.nlm.nih.gov/30358434
https://pubmed.ncbi.nlm.nih.gov/30358434
Autor:
Hari Jayaram, Morgan L. Maeder, Ari E. Friedland, Alex Sousa, Sebastian Gloskowski, G. Grant Welstead, David Bumcrot, McKensie Collins
Publikováno v:
Molecular Therapy. 23
The CRISPR/Cas9 system that is broadly used for genome editing in myriad model organisms and cell lines is derived from the bacterial species Streptoccocus pyogenes. Here we report the use and characterization of another Type II CRISPR endonuclease C
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9e675a6d71e6c6d6e72375883d17425d
Autor:
Morgan L. Maeder, Rina Mepani, Sebastian Gloskowski, Maxwell Skor, McKensie Collins, Shen Shen, Gregory Gotta, Eugenio Marco, Luis Barrera, Hari Jayaram, David Bumcrot
Publikováno v:
Molecular Therapy. 24:S51-S52
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::dc3f81fd8b416b51c5887851da75c274
https://doi.org/10.1016/s1525-0016(16)32933-1
https://doi.org/10.1016/s1525-0016(16)32933-1
Autor:
Hari Jayaram, Rina Mepani, Budd A. Tucker, David Bumcrot, Shen Shen, Sebastian Gloskowski, Morgan L. Maeder, Greg Gotta, Erin R Burnight, Ari E. Friedland
Publikováno v:
Molecular Therapy. 23:S273-S274
Leber congenital amaurosis (LCA) comprises a genetically heterogeneous group of early-onset retinal disorders characterized by severe loss of vision in the first years of life. Approximately 30% of LCA patients harbor mutations in the CEP290 gene, wh
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::15f228273a37484895a04260591dc6b4
https://doi.org/10.1016/s1525-0016(16)34296-4
https://doi.org/10.1016/s1525-0016(16)34296-4