Výsledky vyhledávání - "Aylin C Bonifacino"

Akademický článek

Pre-existing immunity does not impair the engraftment of CRISPR-Cas9-edited cells in rhesus macaques conditioned with busulfan or radiation

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

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Akademický článek

Forced enhancer-promoter rewiring to alter gene expression in animal models

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

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Akademický článek

CD3-immunotoxin mediated depletion of T cells in lymphoid tissues of rhesus macaques

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

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Akademický článek

Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders

Autor: Naoya Uchida, Matthew M. Hsieh, Lydia Raines, Juan J. Haro-Mora, Selami Demirci, Aylin C. Bonifacino, Allen E. Krouse, Mark E. Metzger, Robert E. Donahue, John F. Tisdale

Publikováno v: Nature Communications, Vol 10, Iss 1, Pp 1-14 (2019)

Vectors used in gene therapy for hemoglobin disorders carry globin in a reverse-orientation to prevent the loss of key regulatory elements by RNA splicing, but this limits their efficiency. Here, the authors develop a vector carrying β-globin in a f

Externí odkaz: https://doaj.org/article/10b9f0823d304489858931b8d82f9d3e

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Akademický článek

Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models

Autor: Naoya Uchida, Linhong Li, Tina Nassehi, Claire M. Drysdale, Morgan Yapundich, Jackson Gamer, Juan J. Haro-Mora, Selami Demirci, Alexis Leonard, Aylin C. Bonifacino, Allen E. Krouse, N. Seth Linde, Cornell Allen, Madhusudan V. Peshwa, Suk See De Ravin, Robert E. Donahue, Harry L. Malech, John F. Tisdale

Publikováno v: Cell Reports Medicine, Vol 2, Iss 4, Pp 100247- (2021)

Summary: Sickle cell disease (SCD) is caused by a 20A > T mutation in the β-globin gene. Genome-editing technologies have the potential to correct the SCD mutation in hematopoietic stem cells (HSCs), producing adult hemoglobin while simultaneously e

Externí odkaz: https://doaj.org/article/8e26d04f631d48acaf229ebbbd2bce17

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Comparative engraftment and clonality of macaque HSPCs expanded on human umbilical vein endothelial cells versus non-expanded cells

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)

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

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Akademický článek

Total body irradiation must be delivered at high dose for efficient engraftment and tolerance in a rhesus stem cell gene therapy model

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

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Clonal tracking of erythropoiesis in rhesus macaques

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

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