Zobrazeno 1 - 3
of 3
pro vyhledávání: '"Hoson Chao"'
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:
Charlie Albright, Sandra Teixeira, Jamaica Siwak, Abhishek Dass, Scott Haskett, Kiran Gogi, Diana Tabbaa, Eric L. Tillotson, Terence Ta, Fred Harbinski, Emily Brennan, Gregory Gotta, Ramya Viswanathan, Eugenio Marco, Hoson Chao, Frederick Ta, Deepak Reyon, Jen Da Silva, Meltem Isik, Kai-Hsin Chang, Edouard deDreuzy, Haiyan Jiang, Andrew Sadowski, Georgia Giannoukos, Katherine Loveluck, Tongyao Wang, Aditi Chalishazar, John A. Zuris, Abigail Vogelaar, Ari E. Friedland, Jack Heath, Chris Wilson, Minerva E. Sanchez
Publikováno v:
Blood. 132:409-409
Beta hemoglobinopathies resulting from dysfunctional or deficient adult beta-globin expression are some of the most prevalent inherited blood disorders in the world. Upregulation of developmentally-silenced fetal gamma-globin would replace adult beta
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::6520b0bc5e9bf59489db1d455d8d6922
https://doi.org/10.1182/blood-2018-99-114780
https://doi.org/10.1182/blood-2018-99-114780
Autor:
Volker Schellenberger, Ayman Ismail, Hoson Chao, Sue Patarroyo-White, John Kulman, Ekta Seth Chhabra, Robert T. Peters, Amy M Holthaus, Jiayun Liu, Tongyao Liu, Douglas Drager
Publikováno v:
Blood. 126:3492-3492
All currently marketed Factor VIII (FVIII) molecules are administered intravenously (IV) for the treatment of hemophilia A (HemA). Conventional FVIII prophylaxis requires a dosing interval of three times per week to every other day. This frequent dos
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::6496ebd9e3fd22185bddbf1fb7f64dd9
https://doi.org/10.1182/blood.v126.23.3492.3492
https://doi.org/10.1182/blood.v126.23.3492.3492