Zobrazeno 1 - 10
of 231
pro vyhledávání: '"Naoya Uchida"'
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:
Naoya Uchida, Ulana Stasula, Selami Demirci, Paula Germino-Watnick, Malikiya Hinds, Anh Le, Rebecca Chu, Alexander Berg, Xiong Liu, Ling Su, Xiaolin Wu, Allen E. Krouse, N. Seth Linde, Aylin Bonifacino, So Gun Hong, Cynthia E. Dunbar, Leanne Lanieri, Anjali Bhat, Rahul Palchaudhuri, Bindu Bennet, Megan Hoban, Kirk Bertelsen, Lisa M. Olson, Robert E. Donahue, John F. Tisdale
Publikováno v:
Nature Communications, Vol 14, Iss 1, Pp 1-11 (2023)
Abstract Hematopoietic stem cell (HSC) gene therapy has curative potential; however, its use is limited by the morbidity and mortality associated with current chemotherapy-based conditioning. Targeted conditioning using antibody-drug conjugates (ADC)
Externí odkaz:
https://doaj.org/article/da5777a8fdb54d1094c35c66359052e6
Autor:
Khaled Essawi, Waleed Hakami, Muhammad Behroz Naeem Khan, Reid Martin, Jing Zeng, Rebecca Chu, Naoya Uchida, Aylin C. Bonifacino, Allen E. Krouse, Nathaniel S. Linde, Robert E. Donahue, Gerd A. Blobel, Ulrike Gerdemann, Leslie S. Kean, Stacy A. Maitland, Scot A. Wolfe, Jean-Yves Metais, Stephen Gottschalk, Daniel E. Bauer, John F. Tisdale, Selami Demirci
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 29, Iss , Pp 483-493 (2023)
CRISPR-Cas9-based therapeutic genome editing approaches hold promise to cure a variety of human diseases. Recent findings demonstrate pre-existing immunity for the commonly used Cas orthologs from Streptococcus pyogenes (SpCas9) and Staphylococcus au
Externí odkaz:
https://doaj.org/article/186bb2a612c54f99a14f07e9473590ec
Autor:
Scott A. Peslak, Selami Demirci, Vemika Chandra, Byoung Ryu, Saurabh K. Bhardwaj, Jing Jiang, Jeremy W. Rupon, Robert E. Throm, Naoya Uchida, Alexis Leonard, Khaled Essawi, Aylin C. Bonifacino, Allen E. Krouse, Nathaniel S. Linde, Robert E. Donahue, Francesca Ferrara, Matthew Wielgosz, Osheiza Abdulmalik, Nicole Hamagami, Paula Germino-Watnick, Anh Le, Rebecca Chu, Malikiya Hinds, Mitchell J. Weiss, Wei Tong, John F. Tisdale, Gerd A. Blobel
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 31, Iss , Pp 452-465 (2023)
Transcriptional enhancers can be in physical proximity of their target genes via chromatin looping. The enhancer at the β-globin locus (locus control region [LCR]) contacts the fetal-type (HBG) and adult-type (HBB) β-globin genes during correspondi
Externí odkaz:
https://doaj.org/article/32788d42c8a3431085e1147f913baa4b
Autor:
Naoya Uchida, Claire M. Drysdale, Tina Nassehi, Jackson Gamer, Morgan Yapundich, Julia DiNicola, Yoshitaka Shibata, Malikiya Hinds, Bjorg Gudmundsdottir, Juan J. Haro-Mora, Selami Demirci, John F. Tisdale
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss , Pp 121-132 (2021)
Gene editing with the CRISPR-Cas9 system could revolutionize hematopoietic stem cell (HSC)-targeted gene therapy for hereditary diseases, including sickle cell disease (SCD). Conventional delivery of editing tools by electroporation limits HSC fitnes
Externí odkaz:
https://doaj.org/article/367c1878f8f8434c82d116b980d84a7a
Autor:
Selami Demirci, Bjorg Gudmundsdottir, Quan Li, Juan J. Haro-Mora, Tina Nassehi, Claire Drysdale, Morgan Yapundich, Jackson Gamer, Fayaz Seifuddin, John F. Tisdale, Naoya Uchida
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 912-921 (2020)
Lentiviral addition of βT87Q-globin, a modified β-globin with an anti-sickling mutation, is currently being used in gene therapy trials for sickle cell disease (SCD) and β-thalassemia patients. βT87Q-globin interferes with sickle hemoglobin (HbS)
Externí odkaz:
https://doaj.org/article/b8f50309fcfd40f7925c9851843d83cf
Publikováno v:
Stem Cells Translational Medicine, Vol 9, Iss 5, Pp 590-602 (2020)
Abstract New technologies of induced pluripotent stem cells (iPSCs) and genome editing have emerged, allowing for the development of autologous transfusion therapies. We previously demonstrated definitive β‐globin production from human embryonic s
Externí odkaz:
https://doaj.org/article/9180f58806354d08a4734e42ba2dc3c7
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 16, Iss , Pp 42-49 (2020)
Gene transfer to and correction of hematopoietic stem cells (HSCs) are ideal strategies to cure a number of congenital and acquired disorders. However, transgene products may trigger immunological rejection of modified cells, limiting their therapeut
Externí odkaz:
https://doaj.org/article/888dc8d713e44d1c8ec8224f2fa9ceff
Autor:
Alexis Leonard, Morgan Yapundich, Tina Nassehi, Jackson Gamer, Claire M. Drysdale, Juan J. Haro-Mora, Selami Demirci, Matthew M. Hsieh, Naoya Uchida, John F. Tisdale
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 430-437 (2019)
Humanized animal models are central to efforts aimed at improving hematopoietic stem cell (HSC) transplantation with or without genetic modification. Human cell engraftment is feasible in immunodeficient mice; however, high HSC doses and conditioning
Externí odkaz:
https://doaj.org/article/6db86d5a7bf54e228d1925acc5c5fa51
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