Zobrazeno 1 - 10
of 11
pro vyhledávání: '"Marioara-Felicia Ciuculescu"'
Autor:
Jing Zeng, My Anh Nguyen, Pengpeng Liu, Lucas Ferreira da Silva, Linda Y. Lin, David G. Justus, Karl Petri, Kendell Clement, Shaina N. Porter, Archana Verma, Nola R. Neri, Tolulope Rosanwo, Marioara-Felicia Ciuculescu, Daniela Abriss, Esther Mintzer, Stacy A. Maitland, Selami Demirci, John F. Tisdale, David A. Williams, Lihua Julie Zhu, Shondra M. Pruett-Miller, Luca Pinello, J. Keith Joung, Vikram Pattanayak, John P. Manis, Myriam Armant, Danilo Pellin, Christian Brendel, Scot A. Wolfe, Daniel E. Bauer
Publikováno v:
bioRxiv
SUMMARYGene editing theBCL11Aerythroid enhancer is a validated approach to fetal hemoglobin (HbF) induction for β-hemoglobinopathy therapy, though heterogeneity in edit allele distribution and HbF response may impact its safety and efficacy. Here we
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::250458872ad1c0dd4644117680ac337a
https://doi.org/10.1101/2023.05.27.542323
https://doi.org/10.1101/2023.05.27.542323
Autor:
Erica B. Esrick, Amy Federico, Daniela Abriss, Myriam Armant, Kari Boardman, Christian Brendel, Marioara-Felicia Ciuculescu, Heather Daley, Colleen Dansereau, Augustine Fernandes, Matthew M. Heeney, David G. Justus, Pei-Chi Kao, Annette S. Kim, Donald B. Kohn, Leslie E. Lehmann, Donghui Liu, Wendy B. London, John P Manis, Theodore B. Moore, Emily Morris, Danilo Pellin, Ellen Proeung, Shanna Richard, Gavin D. Roach, Kit L. Shaw, Dayna Terrazas, Shanna L White, David A. Williams
Publikováno v:
Blood. 140:10665-10667
Autor:
Samuele Cancellieri, Jing Zeng, Linda Yingqi Lin, Manuel Tognon, My Anh Nguyen, Jiecong Lin, Nicola Bombieri, Stacy A. Maitland, Marioara-Felicia Ciuculescu, Varun Katta, Shengdar Q. Tsai, Myriam Armant, Scot A. Wolfe, Rosalba Giugno, Daniel E. Bauer, Luca Pinello
Publikováno v:
Nat Genet
CRISPR gene editing holds great promise to modify DNA sequences in somatic cells to treat disease. However, standard computational and biochemical methods to predict off-target potential focus on reference genomes. We developed an efficient tool call
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::85a6955ae43d77107bdfe1de09e8c63a
https://hdl.handle.net/11562/1080767
https://hdl.handle.net/11562/1080767
Autor:
Jing Zeng, My Anh Nguyen, Marioara-Felicia Ciuculescu, Esther Mintzer, Pengpeng Liu, Linda Yingqi Lin, Tolulope O Rosanwo, Archana Verma, Nola Neri, Stacy A. Maitland, Alden Richter, David G. Justus, Kendell Clement, Christian Brendel, Luca Pinello, John P Manis, Myriam Armant, Scot A. Wolfe, Daniel E. Bauer
Publikováno v:
Blood. 140:10671-10672
Autor:
Samuele Cancellieri, Jing Zeng, Linda Yingqi Lin, Manuel Tognon, My Anh Nguyen, Jiecong Lin, Nicola Bombieri, Stacy A. Maitland, Marioara-Felicia Ciuculescu, Varun Katta, Shengdar Q. Tsai, Myriam Armant, Scot A. Wolfe, Rosalba Giugno, Daniel E. Bauer, Luca Pinello
Publikováno v:
Università degli Studi di Verona-IRIS
CRISPR gene editing holds great promise to modify somatic genomes to ameliorate disease. In silico prediction of homologous sites coupled with biochemical evaluation of possible genomic off-targets may predict genotoxicity risk of individual gene edi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f6939987b389bedb32c22997633b0d03
https://doi.org/10.1101/2021.05.20.445054
https://doi.org/10.1101/2021.05.20.445054
Autor:
Myriam Armant, Selami Demirci, David R. Williams, Amornrat Tangprasittipap, Stacy Maitland, Pengpeng Liu, Daniel E. Bauer, J. Keith Joung, Karl Petri, Danilo Pellin, Jing Zeng, John F. Tisdale, Linda Yingqi Lin, John P. Manis, Kevin Luk, Daniela Abriss, Scot A. Wolfe, Yuxuan Wu, Christian Brendel, Shengdar Q. Tsai, Luca Pinello, Varun Katta, Jonathan Y. Hsu, Chokdee Vong, Robert E. Donahue, Shondra M. Pruett-Miller, My Anh Nguyen, Khaled Essawi, Naoya Uchida, Duantida Songdej, Shaina N. Porter, Vikram Pattanayak, Suradej Hongeng, Marioara-Felicia Ciuculescu, Esther Mintzer
Publikováno v:
Blood. 138:1852-1852
Targeting the BCL11A erythroid enhancer by gene editing is a promising approach to fetal hemoglobin induction for beta-hemoglobinopathies. HbF levels vary widely among individuals, suggesting potential heterogeneity in HbF responses after therapeutic
Autor:
Samuele Cancellieri, Rosalba Giugno, Francesco Masillo, Nicola Bombieri, Stacy Maitland, Daniel E. Bauer, Myriam Armant, Linda Yingqi Lin, Luca Pinello, Shengdar Q. Tsai, Marioara-Felicia Ciuculescu, Scot A. Wolfe, My Anh Nguyen, Jing Zeng, Varun Katta
Publikováno v:
Blood. 138:3993-3993
CRISPR gene editing holds great promise to modify somatic genomes to ameliorate disease. In silico prediction of homologous sites coupled with biochemical evaluation of possible genomic off-targets may predict genotoxicity risk of individual gene edi
Autor:
Emily Mitchell, David A. Williams, Grace Boyd, David G. Kent, Myriam Armant, Peter J. Campbell, Alyssa Helene Cull, Michael Spencer Chapman, Marioara Felicia Ciuculescu, Erica B. Esrick
Publikováno v:
Blood. 138:559-559
Recent advances in clonal stem cell tracking strategies have enabled interrogation of unperturbed human hematopoiesis. Whole genome sequencing (WGS) can be used to map the clonal dynamics of hematopoietic stem and progenitor cells (HSPCs) by employin
Autor:
Pablo Bartolucci, Christian Brendel, John M. Higgins, Myriam Armant, David A. Williams, Nicolas Hebert, Etienne Audureau, Erica B. Esrick, Marioara Felicia Ciuculescu
Publikováno v:
Blood. 138:967-967
NH and EE equally contributed. ADW and PB co-signed. The expression of fetal hemoglobin (HbF) is one of the main targets of sickle cell disease treatment, as it inhibits the polymerization of hemoglobin S. The hypothesis of an inhibitory threshold of
Autor:
Erica B. Esrick, Ethan Schonbrun, David K. Wood, Pablo Bartolucci, David R. Williams, John M. Higgins, Marioara Felicia Ciuculescu, Nicolas Hebert, Christian Brendel, Giuseppe Di Caprio, Bronner P. Gonçalves, Emily Morris, Myriam Armant, Daniel C. De Souza
Publikováno v:
Blood. 138:964-964
Introduction Fetal hemoglobin (HbF) induction is a successful strategy for treating sickle cell disease. Higher levels of total HbF% are associated with better clinical outcomes, and a more uniform distribution of HbF across individual red blood cell