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  1. Article ; Online: Intrathecal gene therapy for neurologic disease in humans.

    Flotte, Terence R

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

    2024  

    Language English
    Publishing date 2024-04-24
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1016/j.ymthe.2024.04.013
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 5640: Show issues Location:
    Je nach Verfügbarkeit (siehe Angabe bei Bestand)
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    Jg. 1995 - 2021: Lesesall (2.OG)
    ab Jg. 2022: Lesesaal (EG)

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  2. Article ; Online: Annual Editor's Letter.

    Flotte, Terence R

    Human gene therapy

    2023  Volume 34, Issue 21-22, Page(s) 1073

    Language English
    Publishing date 2023-10-19
    Publishing country United States
    Document type Letter
    ZDB-ID 1028152-6
    ISSN 1557-7422 ; 1043-0342
    ISSN (online) 1557-7422
    ISSN 1043-0342
    DOI 10.1089/hum.2023.29254.letter
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 2988: Show issues Location:
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  3. Article ; Online: Special Editorial Feature: Learning from Our Failures.

    Flotte, Terence R

    Human gene therapy

    2023  Volume 34, Issue 19-20, Page(s) 975

    Language English
    Publishing date 2023-10-12
    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.2023.29246.editorial
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  4. Article ; Online: Lived Experience with Gene Therapy.

    Flotte, Terence R

    Human gene therapy

    2023  Volume 34, Issue 23-24, Page(s) 1178–1179

    Language English
    Publishing date 2023-12-18
    Publishing country United States
    Document type News
    ZDB-ID 1028152-6
    ISSN 1557-7422 ; 1043-0342
    ISSN (online) 1557-7422
    ISSN 1043-0342
    DOI 10.1089/hum.2023.29262.trf
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article ; Online: ESGCT 2022: The New Normal for the Gene Therapy Community.

    Flotte, Terence R

    Human gene therapy

    2022  Volume 33, Issue 17-18, Page(s) 841

    MeSH term(s) Community Health Services ; Genetic Therapy ; Humans
    Language English
    Publishing date 2022-09-20
    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.2022.29217.editorial
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  6. Article ; Online: Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders.

    Daci, Rrita / Flotte, Terence R

    International journal of molecular sciences

    2024  Volume 25, Issue 2

    Abstract: Genetic disorders of the central nervous system (CNS) comprise a significant portion of disability in both children and adults. Several preclinical animal models have shown effective adeno-associated virus (AAV) mediated gene transfer for either ... ...

    Abstract Genetic disorders of the central nervous system (CNS) comprise a significant portion of disability in both children and adults. Several preclinical animal models have shown effective adeno-associated virus (AAV) mediated gene transfer for either treatment or prevention of autosomal recessive genetic disorders. Owing to the intricacy of the human CNS and the blood-brain barrier, it is difficult to deliver genes, particularly since the expression of any given gene may be required in a particular CNS structure or cell type at a specific time during development. In this review, we analyzed delivery methods for AAV-mediated gene therapy in past and current clinical trials. The delivery routes analyzed were direct intraparenchymal (IP), intracerebroventricular (ICV), intra-cisterna magna (CM), lumbar intrathecal (IT), and intravenous (IV). The results demonstrated that the dose used in these routes varies dramatically. The average total doses used were calculated and were 1.03 × 10
    MeSH term(s) Adult ; Animals ; Child ; Infant ; Humans ; Dependovirus/genetics ; Central Nervous System ; Blood-Brain Barrier ; Administration, Intravenous ; Genetic Therapy
    Language English
    Publishing date 2024-01-15
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2019364-6
    ISSN 1422-0067 ; 1422-0067 ; 1661-6596
    ISSN (online) 1422-0067
    ISSN 1422-0067 ; 1661-6596
    DOI 10.3390/ijms25021050
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  7. Article ; Online: ESGCT 2021: Virtually Pan-European.

    Flotte, Terence R

    Human gene therapy

    2021  Volume 32, Issue 19-20, Page(s) 978

    Language English
    Publishing date 2021-10-18
    Publishing country United States
    Document type Journal Article
    ZDB-ID 1028152-6
    ISSN 1557-7422 ; 1043-0342
    ISSN (online) 1557-7422
    ISSN 1043-0342
    DOI 10.1089/hum.2021.29174.trf
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  8. Article ; Online: Base Editing to the Rescue.

    Flotte, Terence R

    Human gene therapy

    2021  Volume 32, Issue 13-14, Page(s) 647–648

    MeSH term(s) CRISPR-Cas Systems/genetics ; Gene Editing
    Language English
    Publishing date 2021-07-20
    Publishing country United States
    Document type Journal Article
    ZDB-ID 1028152-6
    ISSN 1557-7422 ; 1043-0342
    ISSN (online) 1557-7422
    ISSN 1043-0342
    DOI 10.1089/hum.2021.29168.trf
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  9. Article ; Online: Liver targeting with rAAV7: balancing tropism with immune profiles.

    Flotte, Terence R

    Gene therapy

    2021  Volume 28, Issue 3-4, Page(s) 115–116

    MeSH term(s) Dependovirus/genetics ; Liver ; Transduction, Genetic ; Tropism
    Language English
    Publishing date 2021-02-05
    Publishing country England
    Document type Journal Article
    ZDB-ID 1191036-7
    ISSN 1476-5462 ; 0969-7128
    ISSN (online) 1476-5462
    ISSN 0969-7128
    DOI 10.1038/s41434-021-00230-4
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  10. Article ; Online: Science Policy and Funding Implications of the 2020 U.S. Election.

    Flotte, Terence R

    Human gene therapy

    2021  Volume 31, Issue 23-24, Page(s) 1215–1216

    MeSH term(s) COVID-19 ; Health Policy ; Humans ; Politics ; Science/trends ; United States
    Language English
    Publishing date 2021-01-21
    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.2020.29141.trf
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