Zobrazeno 1 - 10
of 78
pro vyhledávání: '"Aylin C Bonifacino"'
Autor:
Naoya Uchida, R Patrick Weitzel, Anna Shvygin, Luke P Skala, Lydia Raines, Aylin C Bonifacino, Allen E Krouse, Mark E Metzger, Robert E Donahue, John F Tisdale
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Reduced intensity conditioning (RIC) is desirable for hematopoietic stem cell (HSC) gene therapy applications. However, low gene marking was previously observed in gene therapy trials, suggesting that RIC might be insufficient for (i) opening niches
Externí odkaz:
https://doaj.org/article/b2866a0cb9114dddbfe8675ea28d0e4a
Autor:
Naoya Uchida, Molly E Evans, Matthew M Hsieh, Aylin C Bonifacino, Allen E Krouse, Mark E Metzger, Stephanie E Sellers, Cynthia E Dunbar, Robert E Donahue, John F Tisdale
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 2, Iss C (2013)
Hematopoietic stem cell (HSC) gene therapy using integrating vectors has a potential leukemogenic risk due to insertional mutagenesis. To reduce this risk, a limitation of ≤2 average vector copy number (VCN) per cell is generally accepted. We devel
Externí odkaz:
https://doaj.org/article/9299f1f68c0e4f92aaa63be0d4ba2ab5
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:
Lan Wang, Gajendra W. Suryawanshi, Shihyoung Kim, Xin Guan, Aylin C. Bonifacino, Mark E. Metzger, Robert E. Donahue, Sanggu Kim, Irvin S.Y. Chen
Publikováno v:
Heliyon, Vol 9, Iss 9, Pp e19435- (2023)
Selective T-cell depletion prior to cell or organ transplantation is considered a preconditioning regimen to induce tolerance and immunosuppression. An immunotoxin consisting of a recombinant anti-CD3 antibody conjugated with diphtheria toxin was use
Externí odkaz:
https://doaj.org/article/6d1b05ec6891474793ee47b9bea7ce1b
Autor:
Samson J. Koelle, Idalia M. Yabe, Michael Ginsberg, Diego A. Espinoza, Chuanfeng Wu, Kyung-Rok Yu, Adam Glaser, So Gun Hong, Lauren L. Truitt, Mark E. Metzger, Allen E. Krouse, Sandhya R. Panch, Cynthia E. Dunbar, Stephanie Sellers, Pradeep K. Dagur, Sandeep Kumar Srivastava, Robert E. Donahue, Aylin C. Bonifacino, Daniel J. Nolan
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss, Pp 703-715 (2021)
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss, Pp 703-715 (2021)
Ex vivo hematopoietic stem and progenitor cell (HSPC) expansion platforms are under active development, designed to increase HSPC numbers and thus engraftment ability of allogeneic cord blood grafts or autologous HSPCs for gene therapies. Murine and
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::759590d5a7af67044c39f4f1c32c3005
https://doi.org/10.1016/j.omtm.2021.02.009
https://doi.org/10.1016/j.omtm.2021.02.009
Autor:
Cynthia E. Dunbar, Theresa Engels, Aylin C. Bonifacino, John F. Tisdale, Robert E. Donahue, Robert F. Hoyt, Randall R. Clevenger, Jan K Davidson-Moncada, Naoya Uchida, Kate Stringaris, William G. Telford, Mark E. Metzger, Richard W. Childs, Timothy Hunt, Noriko Sato, Allen E. Krouse, Peter L. Choyke, Robert Reger, Lydia N. Raines, Jeremy Pantin
Publikováno v:
Blood Adv
Intrabone (IB) injection of umbilical cord blood has been proposed as a potential mechanism to improve transplant engraftment and prevent graft failure. However, conventional IB techniques produce low retention of transplanted cells in the marrow. To
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1fc39e2ec02a26ec2d5d8a1de24dea06
https://doi.org/10.1182/bloodadvances.2020003040
https://doi.org/10.1182/bloodadvances.2020003040
Autor:
Yuxuan Wu, Tina Nassehi, Theresa Engels, John F. Tisdale, Aylin C. Bonifacino, Shengdar Q. Tsai, Alexis Leonard, Nathaniel S. Linde, Kevin Luk, Cicera R. Lazzarotto, Robert E. Donahue, Jackson Gamer, Claire M. Drysdale, Jing Zeng, Morgan Yapundich, Selami Demirci, Daniel E. Bauer, Mitchell J. Weiss, Anne H. Shen, Juan J. Haro-Mora, Scot A. Wolfe, Jasmine Bonanno, Allen E. Krouse, Danilo Pellin, Naoya Uchida, Shondra M. Pruett-Miller, Shaina N. Porter
Publikováno v:
J Clin Invest
Gene editing of the erythroid-specific BCL11A enhancer in hematopoietic stem and progenitor cells (HSPCs) from patients with sickle cell disease (SCD) induces fetal hemoglobin (HbF) without detectable toxicity, as assessed by mouse xenotransplant. He
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2336e49f8e34f06aa9e808324814a9d6
https://doi.org/10.1172/jci140189
https://doi.org/10.1172/jci140189
Autor:
Rong Lu, Aylin C. Bonifacino, Yifan Zhou, Robert E. Donahue, Stefan Cordes, Xing Fan, Cynthia E. Dunbar, Stephanie Sellers, Allen E. Krouse, Chuanfeng Wu, Mark E. Metzger, Lauren L. Truitt, Diego A. Espinoza
Publikováno v:
Haematologica
The classical model of hematopoietic hierarchies is being reconsidered on the basis of data from in vitro assays and single cell expression profiling. Recent experiments suggested that the erythroid lineage might differentiate directly from multipote
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::344eca02c9667d16e5b16f52373f1f06
http://europepmc.org/articles/PMC7327626
http://europepmc.org/articles/PMC7327626
Autor:
Katherine R. Calvo, Idalia M. Yabe, Kristin J Hope, Chuanfeng Wu, Xing Fan, Xiaolin Wu, Rong Lu, Aylin C. Bonifacino, Lauren L. Truitt, Stefan Cordes, Di Yang, Robert E. Donahue, Diego A. Espinoza, Cynthia E. Dunbar, So Gun Hong, Suk See DeRavin, Allen E. Krouse, Mark E. Metzger, John F. Tisdale, Harry L. Malech, Selami Demirci, Naoya Uchida
Publikováno v:
Mol Ther
Lentiviral vectors (LVs) are used for delivery of genes into hematopoietic stem and progenitor cells (HSPCs) in clinical trials worldwide. LVs, in contrast to retroviral vectors, are not associated with insertion site-associated malignant clonal expa
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a10495c55f8a461af58b42b1d1323356
https://doi.org/10.1016/j.ymthe.2019.04.003
https://doi.org/10.1016/j.ymthe.2019.04.003