Zobrazeno 1 - 10
of 93
pro vyhledávání: '"Shengdar Q. Tsai"'
Autor:
Alessia Cavazza, Ayal Hendel, Rasmus O. Bak, Paula Rio, Marc Güell, Duško Lainšček, Virginia Arechavala-Gomeza, Ling Peng, Fatma Zehra Hapil, Joshua Harvey, Francisco G. Ortega, Coral Gonzalez-Martinez, Carsten W. Lederer, Kasper Mikkelsen, Giedrius Gasiunas, Nechama Kalter, Manuel A.F.V. Gonçalves, Julie Petersen, Alejandro Garanto, Lluis Montoliu, Marcello Maresca, Stefan E. Seemann, Jan Gorodkin, Loubna Mazini, Rosario Sanchez, Juan R. Rodriguez-Madoz, Noelia Maldonado-Pérez, Torella Laura, Michael Schmueck-Henneresse, Cristina Maccalli, Julian Grünewald, Gloria Carmona, Neli Kachamakova-Trojanowska, Annarita Miccio, Francisco Martin, Giandomenico Turchiano, Toni Cathomen, Yonglun Luo, Shengdar Q. Tsai, Karim Benabdellah
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 34, Iss , Pp 102066- (2023)
The European Cooperation in Science and Technology (COST) is an intergovernmental organization dedicated to funding and coordinating scientific and technological research in Europe, fostering collaboration among researchers and institutions across co
Externí odkaz:
https://doaj.org/article/dff3b1dde38845db9319c7538193d46c
Autor:
Julie Brault, Taylor Liu, Siyuan Liu, Amanda Lawson, Uimook Choi, Nikita Kozhushko, Vera Bzhilyanskaya, Mara Pavel-Dinu, Ronald J. Meis, Michael A. Eckhaus, Sandra S. Burkett, Marita Bosticardo, Benjamin P. Kleinstiver, Luigi D. Notarangelo, Cicera R. Lazzarotto, Shengdar Q. Tsai, Xiaolin Wu, Gary A. Dahl, Matthew H. Porteus, Harry L. Malech, Suk See De Ravin
Publikováno v:
Frontiers in Immunology, Vol 13 (2023)
IntroductionEx vivo gene therapy for treatment of Inborn errors of Immunity (IEIs) have demonstrated significant clinical benefit in multiple Phase I/II clinical trials. Current approaches rely on engineered retroviral vectors to randomly integrate c
Externí odkaz:
https://doaj.org/article/55c1f88429d84741ac3174cb89df6a2b
Publikováno v:
Genome Biology, Vol 22, Iss 1, Pp 1-11 (2021)
Abstract Prime editing is a revolutionary genome-editing technology that can make a wide range of precise edits in DNA. However, designing highly efficient prime editors (PEs) remains challenging. We develop Easy-Prime, a machine learning–based pro
Externí odkaz:
https://doaj.org/article/031d227d56d645bdaffe2144a4bd931b
Autor:
Pan Gao, Qing Lyu, Amr R. Ghanam, Cicera R. Lazzarotto, Gregory A. Newby, Wei Zhang, Mihyun Choi, Orazio J. Slivano, Kevin Holden, John A. Walker, Anastasia P. Kadina, Rob J. Munroe, Christian M. Abratte, John C. Schimenti, David R. Liu, Shengdar Q. Tsai, Xiaochun Long, Joseph M. Miano
Publikováno v:
Genome Biology, Vol 22, Iss 1, Pp 1-21 (2021)
Abstract Background Most single nucleotide variants (SNVs) occur in noncoding sequence where millions of transcription factor binding sites (TFBS) reside. Here, a comparative analysis of CRISPR-mediated homology-directed repair (HDR) versus the recen
Externí odkaz:
https://doaj.org/article/3ae7242cf5e844faa32def1a4de9bd76
Autor:
Jean-Yves Métais, Phillip A. Doerfler, Thiyagaraj Mayuranathan, Daniel E. Bauer, Stephanie C. Fowler, Matthew M. Hsieh, Varun Katta, Sagar Keriwala, Cicera R. Lazzarotto, Kevin Luk, Michael D. Neel, S. Scott Perry, Samuel T. Peters, Shaina N. Porter, Byoung Y. Ryu, Akshay Sharma, Devlin Shea, John F. Tisdale, Naoya Uchida, Scot A. Wolfe, Kaitly J. Woodard, Yuxuan Wu, Yu Yao, Jing Zeng, Shondra Pruett-Miller, Shengdar Q. Tsai, Mitchell J. Weiss
Publikováno v:
Blood Advances, Vol 3, Iss 21, Pp 3379-3392 (2019)
Abstract: Induction of fetal hemoglobin (HbF) via clustered regularly interspaced short palindromic repeats/Cas9–mediated disruption of DNA regulatory elements that repress γ-globin gene (HBG1 and HBG2) expression is a promising therapeutic strate
Externí odkaz:
https://doaj.org/article/d8bdc8c4db524cf3ab90d0385b570174
Autor:
Killian S. Hanlon, Benjamin P. Kleinstiver, Sara P. Garcia, Mikołaj P. Zaborowski, Adrienn Volak, Stefan E. Spirig, Alissa Muller, Alexander A. Sousa, Shengdar Q. Tsai, Niclas E. Bengtsson, Camilla Lööv, Martin Ingelsson, Jeffrey S. Chamberlain, David P. Corey, Martin J. Aryee, J. Keith Joung, Xandra O. Breakefield, Casey A. Maguire, Bence György
Publikováno v:
Nature Communications, Vol 10, Iss 1, Pp 1-11 (2019)
In-depth characterization of adeno-associated virus (AAV)-mediated CRISPR delivery is still lacking. Here, the authors show high levels of integration into Cas9-induced double-strand breaks (DSBs) in therapeutically relevant genes in vivo.
Externí odkaz:
https://doaj.org/article/d463e02a3bdc40c4aca343f4b409877a
Autor:
Yong Cheng, Shengdar Q. Tsai
Publikováno v:
Genome Biology, Vol 19, Iss 1, Pp 1-7 (2018)
Abstract Genome editing holds remarkable promise to transform human medicine as new therapies that can directly address the genetic causes of disease. However, concerns remain about possible undesired biological consequences of genome editors, partic
Externí odkaz:
https://doaj.org/article/f8923aac185d46929e7d0d57540692df
Autor:
Naoya Uchida, Alexis Leonard, David Stroncek, Sandhya R. Panch, Kamille West, Eoghan Molloy, Thomas E. Hughes, Sara Hauffe, Tiffani Taylor, Courtney Fitzhugh, Jane S. Hankins, Megan Wilson, Akshay Sharma, Shengdar Q. Tsai, Mitch J. Weiss, Matthew Hsieh, John F. Tisdale
Publikováno v:
Haematologica, Vol 105, Iss 10 (2020)
Externí odkaz:
https://doaj.org/article/0afc23d4bba94547aea1f753ae035517
Autor:
Julia Klermund, Saar Gill, Toni Cathomen, Aisha A. AlJanahi, Yifan Zhou, Miriam Y. Kim, Kyung-Rok Yu, Bradley Toms, So Gun Hong, David J. Young, Cicera R. Lazzarotto, Xing Fan, Shirley Chen, Stefan Cordes, Geoffroy Andrieux, Shengdar Q. Tsai, Tae-Hoon Shin, Yuesheng Li, Byung-Chul Lee, Ilker Tunc, Cynthia E. Dunbar, Isabel Jabara, Lauren L. Truitt
Publikováno v:
Mol Ther
The programmable nuclease technology CRISPR-Cas9 has revolutionized gene editing in the last decade. Due to the risk of off-target editing, accurate and sensitive methods for off-target characterization are crucial prior to applying CRISPR-Cas9 thera
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a270ea692de14a5bf2342f6b674eb73a
https://doi.org/10.1016/j.ymthe.2021.06.016
https://doi.org/10.1016/j.ymthe.2021.06.016
Autor:
Christiano R. R. Alves, Leillani L. Ha, Rebecca Yaworski, Cicera R. Lazzarotto, Kathleen A. Christie, Aoife Reilly, Ariane Beauvais, Roman M. Doll, Demitri de la Cruz, Casey A. Maguire, Kathryn J. Swoboda, Shengdar Q. Tsai, Rashmi Kothary, Benjamin P. Kleinstiver
Spinal muscular atrophy (SMA) is a devastating neuromuscular disease caused by mutations in theSMN1gene. Despite the development of various therapies, outcomes can remain suboptimal in SMA infants and the duration of such therapies are uncertain.SMN2
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::4eaa7a91ed126da64d6658d42d9a9fde
https://doi.org/10.1101/2023.01.20.524978
https://doi.org/10.1101/2023.01.20.524978
