Cargando…

Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

CRISPR/Cas9-mediated beta-globin (HBB) gene correction of sickle cell disease (SCD) patient-derived hematopoietic stem cells (HSCs) in combination with autologous transplantation represents a recent paradigm in gene therapy. Although several Cas9-based HBB-correction approaches have been proposed, f...

Descripción completa

Detalles Bibliográficos
Autores principales:	Wilkinson, Adam C., Dever, Daniel P., Baik, Ron, Camarena, Joab, Hsu, Ian, Charlesworth, Carsten T., Morita, Chika, Nakauchi, Hiromitsu, Porteus, Matthew H.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7846836/ https://www.ncbi.nlm.nih.gov/pubmed/33514718 http://dx.doi.org/10.1038/s41467-021-20909-x

Ejemplares similares

Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease
por: Park, So Hyun, et al.
Publicado: (2019)

Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting
por: Charlesworth, Carsten T., et al.
Publicado: (2018)

Cas9-AAV6-engineered human mesenchymal stromal cells improved cutaneous wound healing in diabetic mice
por: Srifa, Waracharee, et al.
Publicado: (2020)

Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6
por: Bak, Rasmus O, et al.
Publicado: (2017)

Sox6 Directly Silences Epsilon Globin Expression in Definitive Erythropoiesis
por: Yi, Zanhua, et al.
Publicado: (2006)

Correction: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6
por: Bak, Rasmus O, et al.
Publicado: (2018)

Deficiency of Antioxidative Paraoxonase 2 (Pon2) Leads to Increased Number of Phenotypic LT-HSCs and Disturbed Erythropoiesis
por: Spiecker, Lisa, et al.
Publicado: (2021)

Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease
por: Xu, Liwen, et al.
Publicado: (2023)

Investigation of Cas9 antibodies in the human eye
por: Toral, Marcus A., et al.
Publicado: (2022)

MicroRNA-96 Directly Inhibits γ-Globin Expression in Human Erythropoiesis
por: Azzouzi, Imane, et al.
Publicado: (2011)

CD14+ monocytes repress gamma globin expression at early stages of erythropoiesis
por: Heshusius, Steven, et al.
Publicado: (2021)

Author Correction: Investigation of Cas9 antibodies in the human eye
por: Toral, Marcus A., et al.
Publicado: (2022)

The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells
por: Sharma, Rajiv, et al.
Publicado: (2021)

Immunological barriers to haematopoietic stem cell gene therapy
por: Charlesworth, Carsten T., et al.
Publicado: (2022)

An optimized Sendai viral vector platform for reprogramming to naive pluripotency
por: Charlesworth, Carsten T., et al.
Publicado: (2022)

Role of Macrophages in Sickle Cell Disease Erythrophagocytosis and Erythropoiesis
por: Sesti-Costa, Renata, et al.
Publicado: (2023)

Sickle cell anemia: clinical diversity and beta S-globin haplotypes
por: Loggetto, Sandra Regina
Publicado: (2013)

Nuclease-mediated gene editing by homologous recombination of the human globin locus
por: Voit, Richard A., et al.
Publicado: (2014)

Fetal hemoglobin rescues ineffective erythropoiesis in sickle cell disease
por: El Hoss, Sara, et al.
Publicado: (2020)

Mbd2-CP2c loop drives adult-type globin gene expression and definitive erythropoiesis
por: Kim, Min Young, et al.
Publicado: (2018)

Ca(2+)tapulting HSCs into action
por: Guitart, Amelie V., et al.
Publicado: (2018)

Thalidomide is more efficient than sodium butyrate in enhancing GATA-1 and EKLF gene expression in erythroid progenitors derived from HSCs with β-globin gene mutation
por: Jalali Far, Mohammad Ali, et al.
Publicado: (2016)

CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem Cells
por: Dever, Daniel P., et al.
Publicado: (2019)

Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling
por: De Souza, Daniel C., et al.
Publicado: (2023)

Biochemical and Molecular analysis of the beta-globin gene on Saudi sickle cell anemia
por: Alenzi, Faris Q., et al.
Publicado: (2019)

Frequency of beta S globin gene haplotypes among sickle cell patients in Nigeria
por: Adabale, Abosede, et al.
Publicado: (2021)

Fate tracking reveals differences between Reelin(+) hepatic stellate cells (HSCs) and Desmin(+) HSCs in activation, migration and proliferation
por: Chen, Ning, et al.
Publicado: (2023)

Human induced pluripotent stem cell derived erythroblasts can undergo definitive erythropoiesis and co-express gamma and beta globins
por: Yang, Cheng-Tao, et al.
Publicado: (2014)

Single-Cell Approaches to Deconvolute the Development of HSCs
por: Xiang, Yang, et al.
Publicado: (2021)

Influence of βS-Globin Haplotypes and Hydroxyurea on Arginase I Levels in Sickle Cell Disease
por: Moreira, J. A., et al.
Publicado: (2016)

Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations
por: Woodard, Kaitly J., et al.
Publicado: (2022)

Beta-S globin haplotypes in patients with sickle cell anemia: one approach to understand the diversity in Brazil
por: Okumura, Jéssika Viviani, et al.
Publicado: (2013)

New variants in beta globin gene among the Palestinian refugees with sickle cell disease in Lebanon
por: Moussa, Esraa Y., et al.
Publicado: (2018)

Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease
por: Poletti, Valentina, et al.
Publicado: (2018)

Human cord blood-derived primitive CD34-negative hematopoietic stem cells (HSCs) are myeloid-biased long-term repopulating HSCs
por: Matsuoka, Y, et al.
Publicado: (2015)

Influence of βS-globin haplotypes hydroxyurea on tumor necrosis factor-alpha levels in sickle cell anemia
por: Laurentino, Marília Rocha, et al.
Publicado: (2014)

Common α-globin variants modify hematologic and other clinical phenotypes in sickle cell trait and disease
por: Raffield, Laura M., et al.
Publicado: (2018)

Presence of atypical beta globin (HBB) gene cluster haplotypes in sickle cell anemia patients of India
por: Nongbri, Sandor Rudy L., et al.
Publicado: (2017)

FV Vectors as Alternative Gene Vehicles for Gene Transfer in HSCs
por: Simantirakis, Emmanouil, et al.
Publicado: (2020)

Just a Spoonful of Sugar Helps the HSCs Move ’Round
por: Kent, David G.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_kpsvgasqebchb4p62ffrcelcqk