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  1. Article ; Online: The RNA World: The Best of Times, the Worst of Times.

    VandenDriessche, Thierry

    Human gene therapy

    2021  Volume 32, Issue 19-20, Page(s) 975–977

    MeSH term(s) RNA/genetics
    Chemical Substances RNA (63231-63-0)
    Language English
    Publishing date 2021-10-04
    Publishing country United States
    Document type Editorial
    ZDB-ID 1028152-6
    ISSN 1557-7422 ; 1043-0342
    ISSN (online) 1557-7422
    ISSN 1043-0342
    DOI 10.1089/hum.2021.29173.tvd
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    Zs.A 2988: Show issues Location:
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  2. Article ; Online: Should patients with haemophilia receive gene therapy?

    VandenDriessche, Thierry / Tatsumi, Kohei / Shima, Midori

    The Lancet. Haematology

    2022  Volume 9, Issue 10, Page(s) e722–e723

    MeSH term(s) Genetic Therapy ; Hemophilia A/genetics ; Hemophilia A/therapy ; Humans
    Language English
    Publishing date 2022-09-29
    Publishing country England
    Document type Journal Article
    ISSN 2352-3026
    ISSN (online) 2352-3026
    DOI 10.1016/S2352-3026(22)00300-3
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article ; Online: Hemophilia "A" gene therapy: Lost in translation.

    VandenDriessche, Thierry / Chuah, Marinee K

    Molecular therapy : the journal of the American Society of Gene Therapy

    2022  Volume 30, Issue 12, Page(s) 3508–3509

    MeSH term(s) Humans ; Hemophilia A/genetics ; Hemophilia A/therapy ; Genetic Therapy
    Language English
    Publishing date 2022-11-22
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't ; Comment
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1016/j.ymthe.2022.11.006
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  4. Article ; Online: First hemophilia B gene therapy approved: More than two decades in the making.

    Herzog, Roland W / VandenDriessche, Thierry / Ozelo, Margareth C

    Molecular therapy : the journal of the American Society of Gene Therapy

    2022  Volume 31, Issue 1, Page(s) 1–2

    MeSH term(s) Humans ; Hemophilia B/genetics ; Hemophilia B/therapy ; Hemophilia A/genetics ; Hemophilia A/therapy ; Factor VIII/genetics ; Genetic Therapy ; Factor IX/genetics ; Genetic Vectors
    Chemical Substances Factor VIII (9001-27-8) ; Factor IX (9001-28-9)
    Language English
    Publishing date 2022-12-16
    Publishing country United States
    Document type Editorial
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1016/j.ymthe.2022.12.001
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  5. Article ; Online: Gene and Cell Therapy: Tearing Down Walls.

    VandenDriessche, Thierry

    Human gene therapy

    2018  Volume 29, Issue 10, Page(s) 1071–1073

    MeSH term(s) Cell- and Tissue-Based Therapy/trends ; Genetic Therapy/trends ; Humans
    Language English
    Publishing date 2018-10-28
    Publishing country United States
    Document type Editorial
    ZDB-ID 1028152-6
    ISSN 1557-7422 ; 1043-0342
    ISSN (online) 1557-7422
    ISSN 1043-0342
    DOI 10.1089/hum.2018.29074.tva
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    Zs.A 2988: Show issues Location:
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  6. Article ; Online: Hemophilia Gene Therapy: The End of the Beginning?

    De Wolf, Dries / Singh, Kshitiz / Chuah, Marinee K / VandenDriessche, Thierry

    Human gene therapy

    2023  Volume 34, Issue 17-18, Page(s) 782–792

    Abstract: Extensive preclinical research over the past 30 years has culminated in the recent regulatory approval of several gene therapy products for hemophilia. Based on the efficacy and safety data in a recently conducted phase III clinical trial, ...

    Abstract Extensive preclinical research over the past 30 years has culminated in the recent regulatory approval of several gene therapy products for hemophilia. Based on the efficacy and safety data in a recently conducted phase III clinical trial,
    MeSH term(s) United States ; Adult ; Child ; Humans ; Hemophilia A/genetics ; Hemophilia A/therapy ; Hemophilia B/genetics ; Hemophilia B/therapy ; DNA, Complementary ; Genetic Therapy
    Chemical Substances Valoctocogene Roxaparvovec ; DNA, Complementary
    Language English
    Publishing date 2023-09-06
    Publishing country United States
    Document type Journal Article ; Review ; Research Support, Non-U.S. Gov't
    ZDB-ID 1028152-6
    ISSN 1557-7422 ; 1043-0342
    ISSN (online) 1557-7422
    ISSN 1043-0342
    DOI 10.1089/hum.2023.112
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  7. Article ; Online: First conditional marketing authorization approval in the European Union for hemophilia "A" gene therapy.

    VandenDriessche, Thierry / Pipe, Steven W / Pierce, Glenn F / Kaczmarek, Radoslaw

    Molecular therapy : the journal of the American Society of Gene Therapy

    2022  Volume 30, Issue 11, Page(s) 3335–3336

    MeSH term(s) Humans ; European Union ; Hemophilia A/genetics ; Hemophilia A/therapy ; Drug Approval ; Genetic Therapy ; Marketing
    Language English
    Publishing date 2022-10-19
    Publishing country United States
    Document type Editorial
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1016/j.ymthe.2022.09.020
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  8. Article ; Online: AAV Capsid Engineering: Zooming in on the Target.

    VandenDriessche, Thierry

    Human gene therapy

    2017  Volume 28, Issue 5, Page(s) 373–374

    MeSH term(s) Animals ; Capsid ; Capsid Proteins/genetics ; Dependovirus/genetics ; Genetic Vectors ; Liver ; Macaca mulatta
    Chemical Substances Capsid Proteins
    Language English
    Publishing date 2017-05-12
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 1028152-6
    ISSN 1557-7422 ; 1043-0342
    ISSN (online) 1557-7422
    ISSN 1043-0342
    DOI 10.1089/hum.2017.29042.tva
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 2988: Show issues Location:
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  9. Article ; Online: Universal allogeneic CAR T cells engineered with Sleeping Beauty transposons and CRISPR-CAS9 for cancer immunotherapy.

    Tipanee, Jaitip / Samara-Kuko, Ermira / Gevaert, Thierry / Chuah, Marinee K / VandenDriessche, Thierry

    Molecular therapy : the journal of the American Society of Gene Therapy

    2022  Volume 30, Issue 10, Page(s) 3155–3175

    Abstract: Allogeneic CD19-specific chimeric antigen receptor (CAR) T cells with inactivated donor T cell receptor (TCR) expression can be used as an "off-the-shelf" therapeutic modality for lymphoid malignancies, thus offering an attractive alternative to ... ...

    Abstract Allogeneic CD19-specific chimeric antigen receptor (CAR) T cells with inactivated donor T cell receptor (TCR) expression can be used as an "off-the-shelf" therapeutic modality for lymphoid malignancies, thus offering an attractive alternative to autologous, patient-derived T cells. Current approaches for T cell engineering mainly rely on the use of viral vectors. Here, we optimized and validated a non-viral genetic modification platform based on Sleeping Beauty (SB) transposons delivered with minicircles to express CD19-28z.CAR and CRISPR-Cas9 ribonucleoparticles to inactivate allogeneic TCRs. Efficient TCR gene disruption was achieved with minimal cytotoxicity and with attainment of robust and stable CD19-28z.CAR expression. The CAR T cells were responsive to CD19+ tumor cells with antitumor activities that induced complete tumor remission in NALM6 tumor-bearing mice while significantly reducing TCR alloreactivity and GvHD development. Single CAR signaling induced the similar T cell signaling signatures in TCR-disrupted CAR T cells and control CAR T cells. In contrast, TCR disruption inhibited T cell signaling/protein phosphorylation compared with the control CAR T cells during dual CAR/TCR signaling. This non-viral SB transposon-CRISPR-Cas9 combination strategy serves as an alternative for generating next-generation CD19-specific CAR T while reducing GvHD risk and easing potential manufacturing constraints intrinsic to viral vectors.
    MeSH term(s) Animals ; Antigens, CD19 ; CRISPR-Cas Systems ; Graft vs Host Disease/metabolism ; Hematopoietic Stem Cell Transplantation ; Immunotherapy ; Immunotherapy, Adoptive ; Mice ; Neoplasms/genetics ; Neoplasms/therapy ; Receptors, Antigen, T-Cell/metabolism ; Receptors, Chimeric Antigen/genetics ; Receptors, Chimeric Antigen/metabolism ; T-Lymphocytes
    Chemical Substances Antigens, CD19 ; Receptors, Antigen, T-Cell ; Receptors, Chimeric Antigen
    Language English
    Publishing date 2022-06-16
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1016/j.ymthe.2022.06.006
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  10. Article ; Online: Getting Into the Rhythm With CRISPR.

    VandenDriessche, Thierry / Chuah, Marinee K

    Circulation research

    2018  Volume 123, Issue 8, Page(s) 928–930

    MeSH term(s) CRISPR-Cas Systems ; Clustered Regularly Interspaced Short Palindromic Repeats ; Humans ; Ryanodine Receptor Calcium Release Channel ; Tachycardia, Ventricular
    Chemical Substances Ryanodine Receptor Calcium Release Channel
    Language English
    Publishing date 2018-12-13
    Publishing country United States
    Document type Editorial ; Research Support, Non-U.S. Gov't ; Comment
    ZDB-ID 80100-8
    ISSN 1524-4571 ; 0009-7330 ; 0931-6876
    ISSN (online) 1524-4571
    ISSN 0009-7330 ; 0931-6876
    DOI 10.1161/CIRCRESAHA.118.313876
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