Zobrazeno 1 - 10
of 11
pro vyhledávání: '"Benjamin Vieira"'
Autor:
Samuel Lessard, Pauline Rimmelé, Hui Ling, Kevin Moran, Benjamin Vieira, Yi-Dong Lin, Gaurav Manohar Rajani, Vu Hong, Andreas Reik, Richard Boismenu, Ben Hsu, Michael Chen, Bettina M. Cockroft, Naoya Uchida, John Tisdale, Asif Alavi, Lakshmanan Krishnamurti, Mehrdad Abedi, Isobelle Galeon, David Reiner, Lin Wang, Anne Ramezi, Pablo Rendo, Mark C. Walters, Dana Levasseur, Robert Peters, Timothy Harris, Alexandra Hicks
Publikováno v:
Scientific Reports, Vol 14, Iss 1, Pp 1-18 (2024)
Abstract BIVV003 is a gene-edited autologous cell therapy in clinical development for the potential treatment of sickle cell disease (SCD). Hematopoietic stem cells (HSC) are genetically modified with mRNA encoding zinc finger nucleases (ZFN) that ta
Externí odkaz:
https://doaj.org/article/eeb0c50036ba4eed80ba42d7f6cae1af
Autor:
Sarah Sturtevant, Ayman Ismail, Kunal Desai, Kevin R. Guertin, Wenjing Li, Benjamin Vieira, Alexandra Hicks, David R. Light, Giuseppe Di Caprio, Martin K. Safo, Melanie Demers, Scott Hansen, Bronner P. Gonçalves, John M. Higgins, Faik N. Musayev, Ethan Schonbrun, Dipti Gupta, David K. Wood
Publikováno v:
Blood Adv
Sickle cell disease (SCD) is associated with hemolysis, vascular inflammation, and organ damage. Affected patients experience chronic painful vaso-occlusive events requiring hospitalization. Hypoxia-induced polymerization of sickle hemoglobin S (HbS)
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ad743b03d57330d6494277eb2a4fe02c
https://pubmed.ncbi.nlm.nih.gov/33661300
https://pubmed.ncbi.nlm.nih.gov/33661300
Autor:
Jason A. West, Olivier Danos, Edward J. Rebar, Kai-Hsin Chang, Kai Chen, Fyodor D. Urnov, Andreas Reik, Mingxuan Zhang, Michael C. Holmes, Chao Sun, Benjamin Vieira, Mei Liu, Timothy J. Sullivan, Vu P. Hong, Qianhe Zhou, Siyuan Tan, Sarah Smith, Yingchun Liu, Xiao Yang, Haiyan Jiang
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 4, Iss C, Pp 137-148 (2017)
Molecular Therapy: Methods & Clinical Development, Vol 4, Iss C, Pp 137-148 (2017)
To develop an effective and sustainable cell therapy for sickle cell disease (SCD), we investigated the feasibility of targeted disruption of the BCL11A gene, either within exon 2 or at the GATAA motif in the intronic erythroid-specific enhancer, usi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::80ec1b8cf042a1a80e31f4a3ab93f859
http://europepmc.org/articles/PMC5363298
http://europepmc.org/articles/PMC5363298
Autor:
Benjamin Vieira, Julia E. Brittain, Sarah Sturtevant, Sriram Krishnamoorthy, Dipti Gupta, Timothy Thullen, Levi Makala, Huo Li, Biaoru Li, David R. Light, Betty S. Pace, Ivan Stone, Nancy Moore, William E. Hobbs
Publikováno v:
JCI insight. 2(20)
Sickle cell disease (SCD) results from a point mutation in the β-globin gene forming hemoglobin S (HbS), which polymerizes in deoxygenated erythrocytes, triggering recurrent painful vaso-occlusive crises and chronic hemolytic anemia. Reactivation of
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1cb5635a1ada9604e54d51e3bd850136
https://pubmed.ncbi.nlm.nih.gov/29046485
https://pubmed.ncbi.nlm.nih.gov/29046485
Autor:
Yukio Nakamura, Sriram Krishnamoorthy, Melanie Demers, Sarah Sturtevant, Benjamin Vieira, Dipti Gupta
Publikováno v:
Blood. 134:3559-3559
Sickle cell disease (SCD) is a genetic disorder caused by a missense mutation in the beta-globin gene [Glu6Val] resulting in the formation of HbS (sickle hemoglobin). HbS polymerizes under low oxygen tension causing an array of severe pathophysiologi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::012a5f39fd4bbde3ce8277095aa993a3
https://doi.org/10.1182/blood-2019-127330
https://doi.org/10.1182/blood-2019-127330
Autor:
Melanie Demers, Sarah Sturtevant, Kevin Guertin, Dipti Gupta, Kunal Desai, Benjamin Vieira, Alexandra Hicks, Ayman Ismail, Yukio Nakamura, Bronner Goncalves, Giuseppe Di Caprio, Ethan Schonbrun, Scott Hansen, Martin K. Safo, David K. Wood, John M. Higgins, David R. Light
Publikováno v:
Blood. 134:2260-2260
Dilution of HbS with non-sickling hemoglobin or hemoglobin with increased oxygen affinity is clinically beneficial in sickle cell disease. Aldehydes, including 5-HMF, tucaresol or GBT440, modify the N-terminus of HbS by reversible covalent imine form
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::9d578b804ef5d5c112905dcc877ae17d
https://doi.org/10.1182/blood-2019-128727
https://doi.org/10.1182/blood-2019-128727
Autor:
Pauline Rimmelé, Denny Dang, Benjamin Vieira, Andreas Reik, David W. Gray, Michael C. Holmes, Samuel Lessard, Dana N. Levasseur, Hui Ling, Vu P. Hong, Kevin Moran
Publikováno v:
Blood. 132:2190-2190
Increases in fetal hemoglobin (HbF) are associated with improved clinical outcomes in the inherited hemoglobinopathies. We are developing a novel gene-edited cell therapy to treat patients with sickle cell disease (SCD) and beta-thalassemia (BT) usin
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::68d3eec9088d5ed7c7bb68e5a373ca59
https://doi.org/10.1182/blood-2018-99-116998
https://doi.org/10.1182/blood-2018-99-116998
Publikováno v:
Blood. 132:1074-1074
Sickle cell disease (SCD) is a genetic hemoglobinopathy driven largely by a single codon mutation of the β-globin gene resulting in polymerization of hemoglobin S (HbS). Anti-sickling approaches that involve increasing the oxygen affinity of HbS to
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::dacc6899b43fd8edc979f556133c5947
https://doi.org/10.1182/blood-2018-99-116319
https://doi.org/10.1182/blood-2018-99-116319
Autor:
Robert T. Peters, William E. Hobbs, Sarah Sturtevant, Benjamin Vieira, Dipti Gupta, Yukio Nakamura, Betty S. Pace, David R. Light, Christine Loh, Sriram Krishnamoorthy, Brian Lucas
Publikováno v:
Blood. 128:4840-4840
Sickle Cell Disease (SCD) is caused by a point mutation in the beta- chain of hemoglobin, triggering a complex pathophysiology resulting in recurrent, painful vaso-occlusive events (VOCs) and chronic hemolytic anemia. Induction of fetal hemoglobin (H
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::9856e9d2a6e8f6f3d9a6e94b06881134
https://doi.org/10.1182/blood.v128.22.4840.4840
https://doi.org/10.1182/blood.v128.22.4840.4840
Autor:
Chao Sun, Xiao Yang, Andreas Reik, Edward J. Rebar, Haiyan Jiang, Sarah Smith, Olivier Danos, Timothy J. Sullivan, Ming Zhang, Kai-Hsin Chang, Vu P. Hong, Siyuan Tan, Fyodor D. Urnov, Kai Chen, Benjamin Vieira, Mei Liu
Publikováno v:
Blood. 126:3234-3234
Sickle cell disease (SCD) is one of the most common inherited blood disorders and is caused by a mutation at the adult beta globin gene resulting in substitution of valine for glutamic acid at position 6 in the encoded protein. While SCD can be cured
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::14d74ddc9b309d8cc45ba32af89b8da6
https://doi.org/10.1182/blood.v126.23.3234.3234
https://doi.org/10.1182/blood.v126.23.3234.3234