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  1. Article: Driving CAR-T cells toward solid lung tumors.

    Staal, Frank J T / Avila-Moreno, Federico

    Molecular therapy. Oncology

    2024  Volume 32, Issue 1, Page(s) 200764

    Language English
    Publishing date 2024-02-07
    Publishing country United States
    Document type News
    ISSN 2950-3299
    ISSN 2950-3299
    DOI 10.1016/j.omton.2024.200764
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  2. Article ; Online: The recombinase activating genes: architects of immune diversity during lymphocyte development.

    Braams, Merijn / Pike-Overzet, Karin / Staal, Frank J T

    Frontiers in immunology

    2023  Volume 14, Page(s) 1210818

    Abstract: ... evolution has used gene rearrangements, also known as variable, diversity, and joining gene segment (V(D)J ...

    Abstract The mature lymphocyte population of a healthy individual has the remarkable ability to recognise an immense variety of antigens. Instead of encoding a unique gene for each potential antigen receptor, evolution has used gene rearrangements, also known as variable, diversity, and joining gene segment (V(D)J) recombination. This process is critical for lymphocyte development and relies on recombination-activating genes-1 (RAG1) and RAG2, here collectively referred to as RAG. RAG serves as powerful genome editing tools for lymphocytes and is strictly regulated to prevent dysregulation. However, in the case of dysregulation, RAG has been implicated in cases of cancer, autoimmunity and severe combined immunodeficiency (SCID). This review examines functional protein domains and motifs of RAG, describes advances in our understanding of the function and (dys)regulation of RAG, discuss new therapeutic options, such as gene therapy, for RAG deficiencies, and explore
    MeSH term(s) Homeodomain Proteins/genetics ; Homeodomain Proteins/metabolism ; Recombinases/genetics ; Gene Rearrangement ; Lymphocytes/metabolism ; Genes, RAG-1/genetics
    Chemical Substances Homeodomain Proteins ; Recombinases
    Language English
    Publishing date 2023-07-11
    Publishing country Switzerland
    Document type Journal Article ; Review ; Research Support, Non-U.S. Gov't
    ZDB-ID 2606827-8
    ISSN 1664-3224 ; 1664-3224
    ISSN (online) 1664-3224
    ISSN 1664-3224
    DOI 10.3389/fimmu.2023.1210818
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  3. Article: The quest for the holy grail: overcoming challenges in expanding human hematopoietic stem cells for clinical use.

    Bastani, Sepideh / Staal, Frank J T / Canté-Barrett, Kirsten

    Stem cell investigation

    2023  Volume 10, Page(s) 15

    Abstract: Hematopoietic stem cell (HSC) transplantation has been the golden standard for many hematological disorders. However, the number of HSCs obtained from several sources, including umbilical cord blood (UCB), often is insufficient for transplantation. For ... ...

    Abstract Hematopoietic stem cell (HSC) transplantation has been the golden standard for many hematological disorders. However, the number of HSCs obtained from several sources, including umbilical cord blood (UCB), often is insufficient for transplantation. For decades, maintaining or even expanding HSCs for therapeutic purposes has been a "holy grail" in stem cell biology. Different methods have been proposed to improve the efficiency of cell expansion and enhance homing potential such as co-culture with stromal cells or treatment with specific agents. Recent progress has shown that this is starting to become feasible using serum-free and well-defined media. Some of these protocols to expand HSCs along with genetic modification have been successfully applied in clinical trials and some others are studied in preclinical and clinical studies. However, the main challenges regarding
    Language English
    Publishing date 2023-07-11
    Publishing country China
    Document type Journal Article ; Review
    ZDB-ID 2884645-X
    ISSN 2313-0792 ; 2306-9759
    ISSN (online) 2313-0792
    ISSN 2306-9759
    DOI 10.21037/sci-2023-016
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  4. Article: Cell intrinsic regulation of external hematopoietic stem cell stress.

    Staal, Frank J T

    Stem cell investigation

    2018  Volume 5, Page(s) 16

    Language English
    Publishing date 2018-05-22
    Publishing country China
    Document type Editorial ; Comment
    ZDB-ID 2884645-X
    ISSN 2313-0792 ; 2306-9759
    ISSN (online) 2313-0792
    ISSN 2306-9759
    DOI 10.21037/sci.2018.05.01
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  5. Article ; Online: Advances in gene therapy for inborn errors of immunity.

    Ott de Bruin, Lisa M / Lankester, Arjan C / Staal, Frank J T

    Current opinion in allergy and clinical immunology

    2023  Volume 23, Issue 6, Page(s) 467–477

    Abstract: Purpose of review: Provide an overview of the landmark accomplishments and state of the art of gene therapy for inborn errors of immunity (IEI).: Recent findings: Three decades after the first clinical application of gene therapy for IEI, there is ... ...

    Abstract Purpose of review: Provide an overview of the landmark accomplishments and state of the art of gene therapy for inborn errors of immunity (IEI).
    Recent findings: Three decades after the first clinical application of gene therapy for IEI, there is one market authorized product available, while for several others efficacy has been demonstrated or is currently being tested in ongoing clinical trials. Gene editing approaches using programmable nucleases are being explored preclinically and could be beneficial for genes requiring tightly regulated expression, gain-of-function mutations and dominant-negative mutations.
    Summary: Gene therapy by modifying autologous hematopoietic stem cells (HSCs) offers an attractive alternative to allogeneic hematopoietic stem cell transplantation (HSCT), the current standard of care to treat severe IEI. This approach does not require availability of a suitable allogeneic donor and eliminates the risk of graft versus host disease (GvHD). Gene therapy can be attempted by using a viral vector to add a copy of the therapeutic gene (viral gene addition) or by using programmable nucleases (gene editing) to precisely correct mutations, disrupt a gene or introduce an entire copy of a gene at a specific locus. However, gene therapy comes with its own challenges such as safety, therapeutic effectiveness and access. For viral gene addition, a major safety concern is vector-related insertional mutagenesis, although this has been greatly reduced with the introduction of safer vectors. For gene editing, the risk of off-site mutagenesis is a main driver behind the ongoing search for modified nucleases. For both approaches, HSCs have to be manipulated ex vivo, and doing this efficiently without losing stemness remains a challenge, especially for gene editing.
    MeSH term(s) Humans ; Hematopoietic Stem Cell Transplantation ; Graft vs Host Disease ; Genetic Therapy ; Gene Editing
    Language English
    Publishing date 2023-10-13
    Publishing country United States
    Document type Review ; Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2088710-3
    ISSN 1473-6322 ; 1528-4050
    ISSN (online) 1473-6322
    ISSN 1528-4050
    DOI 10.1097/ACI.0000000000000952
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    In stock of ZB MED Cologne/Königswinter

    Zs.A 5666: Show issues Location:
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  6. Article ; Online: The Route of Early T Cell Development: Crosstalk between Epigenetic and Transcription Factors.

    Chiara, Veronica Della / Daxinger, Lucia / Staal, Frank J T

    Cells

    2021  Volume 10, Issue 5

    Abstract: ... developmental stages until the formation of mature T cells. During this process, phenotypic changes of T cells entail ... Lineage-specific transcription factors are key drivers of T cell specification and act in conjunction ... networks necessary for proper T cell development. In this review, we summarize ...

    Abstract Hematopoietic multipotent progenitors seed the thymus and then follow consecutive developmental stages until the formation of mature T cells. During this process, phenotypic changes of T cells entail stage-specific transcriptional programs that underlie the dynamic progression towards mature lymphocytes. Lineage-specific transcription factors are key drivers of T cell specification and act in conjunction with epigenetic regulators that have also been elucidated as crucial players in the establishment of regulatory networks necessary for proper T cell development. In this review, we summarize the activity of transcription factors and epigenetic regulators that together orchestrate the intricacies of early T cell development with a focus on regulation of T cell lineage commitment.
    MeSH term(s) Animals ; Chromatin Assembly and Disassembly ; DNA Methylation ; Epigenesis, Genetic ; Humans ; Lymphopoiesis ; T-Lymphocytes/cytology ; T-Lymphocytes/metabolism ; Transcription Factors/metabolism
    Chemical Substances Transcription Factors
    Language English
    Publishing date 2021-04-30
    Publishing country Switzerland
    Document type Journal Article ; Research Support, Non-U.S. Gov't ; Review
    ZDB-ID 2661518-6
    ISSN 2073-4409 ; 2073-4409
    ISSN (online) 2073-4409
    ISSN 2073-4409
    DOI 10.3390/cells10051074
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  7. Article: Wnt signalling meets epigenetics.

    Staal, Frank J T

    Stem cell investigation

    2016  Volume 3, Page(s) 38

    Language English
    Publishing date 2016-08-12
    Publishing country China
    Document type Journal Article
    ZDB-ID 2884645-X
    ISSN 2313-0792 ; 2306-9759
    ISSN (online) 2313-0792
    ISSN 2306-9759
    DOI 10.21037/sci.2016.08.01
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  8. Article ; Online: BETting on stem cell expansion.

    Staal, Frank J T / Fibbe, Willem E

    Blood

    2020  Volume 136, Issue 21, Page(s) 2364–2365

    MeSH term(s) Acetamides ; Azepines ; Fetal Blood ; Gambling ; Humans ; Megakaryocytes ; Stem Cells
    Chemical Substances Acetamides ; Azepines ; CPI203
    Language English
    Publishing date 2020-11-19
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 80069-7
    ISSN 1528-0020 ; 0006-4971
    ISSN (online) 1528-0020
    ISSN 0006-4971
    DOI 10.1182/blood.2020007759
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    In stock of ZB MED Cologne/Königswinter

    Uh III Zs.140: Show issues Location:
    Je nach Verfügbarkeit (siehe Angabe bei Bestand)
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  9. Article ; Online: An adequate human T cell repertoire from a single T cell progenitor: Lessons from an experiment of nature.

    Canté-Barrett, Kirsten / Staal, Frank J T

    EBioMedicine

    2020  Volume 60, Page(s) 103015

    MeSH term(s) Animals ; Clonal Hematopoiesis/genetics ; Disease Susceptibility ; Humans ; Lymphopoiesis/genetics ; Precursor Cells, T-Lymphoid/cytology ; Precursor Cells, T-Lymphoid/metabolism ; Receptors, Antigen, T-Cell/genetics ; T-Lymphocytes/cytology ; T-Lymphocytes/metabolism ; V(D)J Recombination
    Chemical Substances Receptors, Antigen, T-Cell
    Language English
    Publishing date 2020-09-22
    Publishing country Netherlands
    Document type Letter
    ZDB-ID 2851331-9
    ISSN 2352-3964
    ISSN (online) 2352-3964
    DOI 10.1016/j.ebiom.2020.103015
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    In stock of ZB MED Cologne/Königswinter

    Zs.A 7090: Show issues Location:
    Je nach Verfügbarkeit (siehe Angabe bei Bestand)
    bis Jg. 2021: Bestellungen von Artikeln über das Online-Bestellformular
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  10. Article ; Online: Corrigendum: Blocking of the High-Affinity Interaction-Synapse Between SARS-CoV-2 Spike and Human ACE2 Proteins Likely Requires Multiple High-Affinity Antibodies: An Immune Perspective.

    Khatri, Indu / Staal, Frank J T / van Dongen, Jacques J M

    Frontiers in immunology

    2021  Volume 12, Page(s) 659375

    Abstract: This corrects the article DOI: 10.3389/fimmu.2020.570018.]. ...

    Abstract [This corrects the article DOI: 10.3389/fimmu.2020.570018.].
    Language English
    Publishing date 2021-04-14
    Publishing country Switzerland
    Document type Published Erratum
    ZDB-ID 2606827-8
    ISSN 1664-3224 ; 1664-3224
    ISSN (online) 1664-3224
    ISSN 1664-3224
    DOI 10.3389/fimmu.2021.659375
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