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  1. Article ; Online: Author Correction: Genotoxic effects of base and prime editing in human hematopoietic stem cells.

    Fiumara, Martina / Ferrari, Samuele / Omer-Javed, Attya / Beretta, Stefano / Albano, Luisa / Canarutto, Daniele / Varesi, Angelica / Gaddoni, Chiara / Brombin, Chiara / Cugnata, Federica / Zonari, Erika / Naldini, Matteo Maria / Barcella, Matteo / Gentner, Bernhard / Merelli, Ivan / Naldini, Luigi

    Nature biotechnology

    2024  

    Language English
    Publishing date 2024-01-25
    Publishing country United States
    Document type Published Erratum
    ZDB-ID 1311932-1
    ISSN 1546-1696 ; 1087-0156
    ISSN (online) 1546-1696
    ISSN 1087-0156
    DOI 10.1038/s41587-024-02142-1
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article: Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells

    Omer-Javed, Attya / Pedrazzani, Gabriele / Albano, Luisa / Ghaus, Sherash / Latroche, Claire / Manzi, Maura / Ferrari, Samuele / Fiumara, Martina / Jacob, Aurelien / Vavassori, Valentina / Nonis, Alessandro / Canarutto, Daniele / Naldini, Luigi

    Cell. 2022 June 23, v. 185, no. 13

    2022  

    Abstract: Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) is proving successful to treat several genetic diseases. HSPCs are mobilized, harvested, genetically corrected ex vivo, and infused, after the administration of toxic myeloablative conditioning to ...

    Abstract Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) is proving successful to treat several genetic diseases. HSPCs are mobilized, harvested, genetically corrected ex vivo, and infused, after the administration of toxic myeloablative conditioning to deplete the bone marrow (BM) for the modified cells. We show that mobilizers create an opportunity for seamless engraftment of exogenous cells, which effectively outcompete those mobilized, to repopulate the depleted BM. The competitive advantage results from the rescue during ex vivo culture of a detrimental impact of mobilization on HSPCs and can be further enhanced by the transient overexpression of engraftment effectors exploiting optimized mRNA-based delivery. We show the therapeutic efficacy in a mouse model of hyper IgM syndrome and further developed it in human hematochimeric mice, showing its applicability and versatility when coupled with gene transfer and editing strategies. Overall, our findings provide a potentially valuable strategy paving the way to broader and safer use of HSPC-GT.
    Keywords bone marrow ; gene therapy ; gene transfer ; humans ; mice ; toxicity
    Language English
    Dates of publication 2022-0623
    Size p. 2248-2264.e21.
    Publishing place Elsevier Inc.
    Document type Article
    ZDB-ID 187009-9
    ISSN 1097-4172 ; 0092-8674
    ISSN (online) 1097-4172
    ISSN 0092-8674
    DOI 10.1016/j.cell.2022.04.039
    Database NAL-Catalogue (AGRICOLA)

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  3. Article ; Online: Genotoxic effects of base and prime editing in human hematopoietic stem cells.

    Fiumara, Martina / Ferrari, Samuele / Omer-Javed, Attya / Beretta, Stefano / Albano, Luisa / Canarutto, Daniele / Varesi, Angelica / Gaddoni, Chiara / Brombin, Chiara / Cugnata, Federica / Zonari, Erika / Naldini, Matteo Maria / Barcella, Matteo / Gentner, Bernhard / Merelli, Ivan / Naldini, Luigi

    Nature biotechnology

    2023  

    Abstract: Base and prime editors (BEs and PEs) may provide more precise genetic engineering than nuclease-based approaches because they bypass the dependence on DNA double-strand breaks. However, little is known about their cellular responses and genotoxicity. ... ...

    Abstract Base and prime editors (BEs and PEs) may provide more precise genetic engineering than nuclease-based approaches because they bypass the dependence on DNA double-strand breaks. However, little is known about their cellular responses and genotoxicity. Here, we compared state-of-the-art BEs and PEs and Cas9 in human hematopoietic stem and progenitor cells with respect to editing efficiency, cytotoxicity, transcriptomic changes and on-target and genome-wide genotoxicity. BEs and PEs induced detrimental transcriptional responses that reduced editing efficiency and hematopoietic repopulation in xenotransplants and also generated DNA double-strand breaks and genotoxic byproducts, including deletions and translocations, at a lower frequency than Cas9. These effects were strongest for cytidine BEs due to suboptimal inhibition of base excision repair and were mitigated by tailoring delivery timing and editor expression through optimized mRNA design. However, BEs altered the mutational landscape of hematopoietic stem and progenitor cells across the genome by increasing the load and relative proportions of nucleotide variants. These findings raise concerns about the genotoxicity of BEs and PEs and warrant further investigation in view of their clinical application.
    Language English
    Publishing date 2023-09-07
    Publishing country United States
    Document type Journal Article
    ZDB-ID 1311932-1
    ISSN 1546-1696 ; 1087-0156
    ISSN (online) 1546-1696
    ISSN 1087-0156
    DOI 10.1038/s41587-023-01915-4
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  4. Article ; Online: Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4

    Canarutto, Daniele / Asperti, Claudia / Vavassori, Valentina / Porcellini, Simona / Rovelli, Elisabetta / Paulis, Marianna / Ferrari, Samuele / Varesi, Angelica / Fiumara, Martina / Jacob, Aurelien / Sergi Sergi, Lucia / Visigalli, Ilaria / Ferrua, Francesca / González-Granado, Luis Ignacio / Lougaris, Vassilios / Finocchi, Andrea / Villa, Anna / Radrizzani, Marina / Naldini, Luigi

    The EMBO journal

    2023  Volume 42, Issue 23, Page(s) e114188

    Abstract: Hyper IgM1 is an X-linked combined immunodeficiency caused by CD40LG mutations, potentially treatable with ... ...

    Abstract Hyper IgM1 is an X-linked combined immunodeficiency caused by CD40LG mutations, potentially treatable with CD4
    MeSH term(s) Humans ; CRISPR-Cas Systems ; Gene Editing/methods ; Genome ; T-Lymphocytes ; CD4-Positive T-Lymphocytes
    Language English
    Publishing date 2023-11-02
    Publishing country England
    Document type Journal Article
    ZDB-ID 586044-1
    ISSN 1460-2075 ; 0261-4189
    ISSN (online) 1460-2075
    ISSN 0261-4189
    DOI 10.15252/embj.2023114188
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  5. Article ; Online: Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells.

    Omer-Javed, Attya / Pedrazzani, Gabriele / Albano, Luisa / Ghaus, Sherash / Latroche, Claire / Manzi, Maura / Ferrari, Samuele / Fiumara, Martina / Jacob, Aurelien / Vavassori, Valentina / Nonis, Alessandro / Canarutto, Daniele / Naldini, Luigi

    Cell

    2022  Volume 185, Issue 13, Page(s) 2248–2264.e21

    Abstract: Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) is proving successful to treat several genetic diseases. HSPCs are mobilized, harvested, genetically corrected ex vivo, and infused, after the administration of toxic myeloablative conditioning to ...

    Abstract Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) is proving successful to treat several genetic diseases. HSPCs are mobilized, harvested, genetically corrected ex vivo, and infused, after the administration of toxic myeloablative conditioning to deplete the bone marrow (BM) for the modified cells. We show that mobilizers create an opportunity for seamless engraftment of exogenous cells, which effectively outcompete those mobilized, to repopulate the depleted BM. The competitive advantage results from the rescue during ex vivo culture of a detrimental impact of mobilization on HSPCs and can be further enhanced by the transient overexpression of engraftment effectors exploiting optimized mRNA-based delivery. We show the therapeutic efficacy in a mouse model of hyper IgM syndrome and further developed it in human hematochimeric mice, showing its applicability and versatility when coupled with gene transfer and editing strategies. Overall, our findings provide a potentially valuable strategy paving the way to broader and safer use of HSPC-GT.
    MeSH term(s) Animals ; Gene Editing ; Genetic Therapy/methods ; Hematopoietic Stem Cell Transplantation/methods ; Hematopoietic Stem Cells ; Humans ; Mice
    Language English
    Publishing date 2022-05-25
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 187009-9
    ISSN 1097-4172 ; 0092-8674
    ISSN (online) 1097-4172
    ISSN 0092-8674
    DOI 10.1016/j.cell.2022.04.039
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  6. Article: Metformin reduces the clonal fitness of

    Hosseini, Mohsen / Voisin, Veronique / Chegini, Ali / Varesi, Angelica / Cathelin, Severine / Ayyathan, Dhanoop Manikoth / Liu, Alex C H / Yang, Yitong / Wang, Vivian / Maher, Abdula / Grignano, Eric / Reisz, Julie A / D'Alessandro, Angelo / Young, Kira / Wu, Yiyan / Fiumara, Martina / Ferrari, Samuele / Naldini, Luigi / Gaiti, Federico /
    Pai, Shraddha / Schimmer, Aaron D / Bader, Gary D / Dick, John E / Xie, Stephanie Z / Trowbridge, Jennifer J / Chan, Steven M

    Research square

    2024  

    Abstract: Clonal hematopoiesis (CH) arises when a hematopoietic stem cell (HSC) acquires a mutation that confers a competitive advantage over wild-type (WT) HSCs, resulting in its clonal expansion. Individuals with CH are at an increased risk of developing ... ...

    Abstract Clonal hematopoiesis (CH) arises when a hematopoietic stem cell (HSC) acquires a mutation that confers a competitive advantage over wild-type (WT) HSCs, resulting in its clonal expansion. Individuals with CH are at an increased risk of developing hematologic neoplasms and a range of age-related inflammatory illnesses
    Language English
    Publishing date 2024-02-06
    Publishing country United States
    Document type Preprint
    DOI 10.21203/rs.3.rs-3874821/v1
    Database MEDical Literature Analysis and Retrieval System OnLINE

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