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  1. Article ; Online: SGK1 inhibition induces fetal hemoglobin expression and delays polymerization in sickle erythroid cells.

    Hara, Yannis / Lemgart, Viktor T / Halland, Nis / Mahdaviani, Kiana / Ribeil, Jean-Antoine / Lessard, Samuel / Hicks, Alexandra / Light, David R

    Blood advances

    2023  Volume 7, Issue 11, Page(s) 2317–2323

    MeSH term(s) Humans ; Fetal Hemoglobin/genetics ; Fetal Hemoglobin/metabolism ; Polymerization ; Erythroid Cells/metabolism ; Anemia, Sickle Cell/metabolism ; Erythrocytes, Abnormal/metabolism
    Chemical Substances Fetal Hemoglobin (9034-63-3)
    Language English
    Publishing date 2023-01-25
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2915908-8
    ISSN 2473-9537 ; 2473-9529
    ISSN (online) 2473-9537
    ISSN 2473-9529
    DOI 10.1182/bloodadvances.2022008710
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Zs.A 7153: Show issues Location:
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  2. Article ; Online: Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells.

    Cromer, M Kyle / Camarena, Joab / Martin, Renata M / Lesch, Benjamin J / Vakulskas, Christopher A / Bode, Nicole M / Kurgan, Gavin / Collingwood, Michael A / Rettig, Garrett R / Behlke, Mark A / Lemgart, Viktor T / Zhang, Yankai / Goyal, Ankush / Zhao, Feifei / Ponce, Ezequiel / Srifa, Waracharee / Bak, Rasmus O / Uchida, Naoya / Majeti, Ravindra /
    Sheehan, Vivien A / Tisdale, John F / Dever, Daniel P / Porteus, Matthew H

    Nature medicine

    2021  Volume 27, Issue 4, Page(s) 677–687

    Abstract: β-Thalassemia pathology is due not only to loss of β-globin (HBB), but also to erythrotoxic accumulation and aggregation of the β-globin-binding partner, α-globin (HBA1/2). Here we describe a Cas9/AAV6-mediated genome editing strategy that can replace ... ...

    Abstract β-Thalassemia pathology is due not only to loss of β-globin (HBB), but also to erythrotoxic accumulation and aggregation of the β-globin-binding partner, α-globin (HBA1/2). Here we describe a Cas9/AAV6-mediated genome editing strategy that can replace the entire HBA1 gene with a full-length HBB transgene in β-thalassemia-derived hematopoietic stem and progenitor cells (HSPCs), which is sufficient to normalize β-globin:α-globin messenger RNA and protein ratios and restore functional adult hemoglobin tetramers in patient-derived red blood cells. Edited HSPCs were capable of long-term and bilineage hematopoietic reconstitution in mice, establishing proof of concept for replacement of HBA1 with HBB as a novel therapeutic strategy for curing β-thalassemia.
    MeSH term(s) Anemia, Sickle Cell/pathology ; Animals ; Antigens, CD34/metabolism ; Dependovirus/genetics ; Erythrocytes/metabolism ; Gene Editing ; Genes, Reporter ; Genetic Loci ; Genetic Therapy ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells/metabolism ; Hemoglobins/metabolism ; Humans ; Mice ; Promoter Regions, Genetic/genetics ; alpha-Globins/genetics ; beta-Globins/genetics ; beta-Thalassemia/genetics ; beta-Thalassemia/therapy
    Chemical Substances Antigens, CD34 ; Hemoglobins ; alpha-Globins ; beta-Globins
    Language English
    Publishing date 2021-03-18
    Publishing country United States
    Document type Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
    ZDB-ID 1220066-9
    ISSN 1546-170X ; 1078-8956
    ISSN (online) 1546-170X
    ISSN 1078-8956
    DOI 10.1038/s41591-021-01284-y
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 4212: Show issues Location:
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  3. Article ; Online: Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination.

    Martin, Renata M / Ikeda, Kazuya / Cromer, M Kyle / Uchida, Nobuko / Nishimura, Toshinobu / Romano, Rosa / Tong, Andrew J / Lemgart, Viktor T / Camarena, Joab / Pavel-Dinu, Mara / Sindhu, Camille / Wiebking, Volker / Vaidyanathan, Sriram / Dever, Daniel P / Bak, Rasmus O / Laustsen, Anders / Lesch, Benjamin J / Jakobsen, Martin R / Sebastiano, Vittorio /
    Nakauchi, Hiromitsu / Porteus, Matthew H

    Cell stem cell

    2019  Volume 24, Issue 5, Page(s) 821–828.e5

    Abstract: Genome editing of human pluripotent stem cells (hPSCs) provides powerful opportunities for in vitro disease modeling, drug discovery, and personalized stem cell-based therapeutics. Currently, only small edits can be engineered with high frequency, while ... ...

    Abstract Genome editing of human pluripotent stem cells (hPSCs) provides powerful opportunities for in vitro disease modeling, drug discovery, and personalized stem cell-based therapeutics. Currently, only small edits can be engineered with high frequency, while larger modifications suffer from low efficiency and a resultant need for selection markers. Here, we describe marker-free genome editing in hPSCs using Cas9 ribonucleoproteins (RNPs) in combination with AAV6-mediated DNA repair template delivery. We report highly efficient and bi-allelic integration frequencies across multiple loci and hPSC lines, achieving mono-allelic editing frequencies of up to 94% at the HBB locus. Using this method, we show robust bi-allelic correction of homozygous sickle cell mutations in a patient-derived induced PSC (iPSC) line. Thus, this strategy shows significant utility for generating hPSCs with large gene integrations and/or single-nucleotide changes at high frequency and without the need for introducing selection genes, enhancing the applicability of hPSC editing for research and translational uses.
    MeSH term(s) CRISPR-Associated Protein 9/genetics ; CRISPR-Associated Protein 9/metabolism ; CRISPR-Cas Systems/genetics ; Clustered Regularly Interspaced Short Palindromic Repeats ; DNA Repair ; Dependovirus/genetics ; Gene Editing/methods ; Gene Frequency ; Genetic Engineering ; Genetic Vectors/genetics ; Genotype ; Homologous Recombination ; Humans ; Pathology, Molecular ; Pluripotent Stem Cells/physiology ; Tissue Donors
    Chemical Substances CRISPR-Associated Protein 9 (EC 3.1.-)
    Language English
    Publishing date 2019-05-01
    Publishing country United States
    Document type Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
    ZDB-ID 2375354-7
    ISSN 1875-9777 ; 1934-5909
    ISSN (online) 1875-9777
    ISSN 1934-5909
    DOI 10.1016/j.stem.2019.04.001
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  4. Article ; Online: Identification of preexisting adaptive immunity to Cas9 proteins in humans.

    Charlesworth, Carsten T / Deshpande, Priyanka S / Dever, Daniel P / Camarena, Joab / Lemgart, Viktor T / Cromer, M Kyle / Vakulskas, Christopher A / Collingwood, Michael A / Zhang, Liyang / Bode, Nicole M / Behlke, Mark A / Dejene, Beruh / Cieniewicz, Brandon / Romano, Rosa / Lesch, Benjamin J / Gomez-Ospina, Natalia / Mantri, Sruthi / Pavel-Dinu, Mara / Weinberg, Kenneth I /
    Porteus, Matthew H

    Nature medicine

    2019  Volume 25, Issue 2, Page(s) 249–254

    Abstract: The CRISPR-Cas9 system is a powerful tool for genome editing, which allows the precise modification of specific DNA sequences. Many efforts are underway to use the CRISPR-Cas9 system to therapeutically correct human genetic ... ...

    Abstract The CRISPR-Cas9 system is a powerful tool for genome editing, which allows the precise modification of specific DNA sequences. Many efforts are underway to use the CRISPR-Cas9 system to therapeutically correct human genetic diseases
    MeSH term(s) Adaptive Immunity ; Adult ; CRISPR-Associated Protein 9/metabolism ; Cell Separation ; Female ; Humans ; Immunity, Humoral ; Male ; T-Lymphocytes/immunology
    Chemical Substances CRISPR-Associated Protein 9 (EC 3.1.-)
    Language English
    Publishing date 2019-01-28
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 1220066-9
    ISSN 1546-170X ; 1078-8956
    ISSN (online) 1546-170X
    ISSN 1078-8956
    DOI 10.1038/s41591-018-0326-x
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 4212: Show issues Location:
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