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  1. Article ; Online: In memoriam: Kenneth I. Berns, MD, PhD (1938-2024).

    Byrne, Barry J / Flotte, Terence R / Herzog, Roland W / Srivastava, Arun

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

    2024  Volume 32, Issue 4, Page(s) 867–868

    Language English
    Publishing date 2024-03-06
    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.2024.02.018
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Zs.A 5640: Show issues Location:
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  2. Article ; Online: Neuromuscular Gene Therapy: Catching the Wave of Positive Early Studies Is Expected to Crest in Product Approvals.

    Byrne, Barry J

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

    2019  Volume 27, Issue 10, Page(s) 1695–1696

    MeSH term(s) Dependovirus/genetics ; Drug Approval ; Genetic Therapy/methods ; Genetic Vectors/administration & dosage ; Humans ; Neuromuscular Diseases/genetics ; Neuromuscular Diseases/therapy
    Language English
    Publishing date 2019-09-23
    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.2019.09.011
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 5640: Show issues Location:
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  3. Article ; Online: Kenneth I. Berns, MD, PhD [1938-2024].

    Byrne, Barry J / Flotte, Terence R / Herzog, Roland W / Srivastava, Arun

    Human gene therapy

    2024  Volume 35, Issue 5-6, Page(s) 133–134

    Language English
    Publishing date 2024-03-18
    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.2024.29266.kib
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 2988: Show issues Location:
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  4. Article ; Online: Evolving Horizons: Adenovirus Vectors' Timeless Influence on Cancer, Gene Therapy and Vaccines.

    Trivedi, Prasad D / Byrne, Barry J / Corti, Manuela

    Viruses

    2023  Volume 15, Issue 12

    Abstract: Efficient and targeted delivery of a DNA payload is vital for developing safe gene therapy. Owing to the recent success of commercial oncolytic vector and multiple COVID-19 vaccines, adenovirus vectors are back in the spotlight. Adenovirus vectors can be ...

    Abstract Efficient and targeted delivery of a DNA payload is vital for developing safe gene therapy. Owing to the recent success of commercial oncolytic vector and multiple COVID-19 vaccines, adenovirus vectors are back in the spotlight. Adenovirus vectors can be used in gene therapy by altering the wild-type virus and making it replication-defective; specific viral genes can be removed and replaced with a segment that holds a therapeutic gene, and this vector can be used as delivery vehicle for tissue specific gene delivery. Modified conditionally replicative-oncolytic adenoviruses target tumors exclusively and have been studied in clinical trials extensively. This comprehensive review seeks to offer a summary of adenovirus vectors, exploring their characteristics, genetic enhancements, and diverse applications in clinical and preclinical settings. A significant emphasis is placed on their crucial role in advancing cancer therapy and the latest breakthroughs in vaccine clinical trials for various diseases. Additionally, we tackle current challenges and future avenues for optimizing adenovirus vectors, promising to open new frontiers in the fields of cell and gene therapies.
    MeSH term(s) Humans ; COVID-19 Vaccines ; Virus Replication/genetics ; Neoplasms/genetics ; Neoplasms/therapy ; Genetic Vectors/genetics ; Adenoviridae/genetics ; Genetic Therapy ; Vaccines
    Chemical Substances COVID-19 Vaccines ; Vaccines
    Language English
    Publishing date 2023-12-03
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2516098-9
    ISSN 1999-4915 ; 1999-4915
    ISSN (online) 1999-4915
    ISSN 1999-4915
    DOI 10.3390/v15122378
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article ; Online: Safety First: Perspective on Patient-Centered Development of AAV Gene Therapy Products.

    Byrne, Barry J

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

    2018  Volume 26, Issue 3, Page(s) 669–671

    MeSH term(s) Administration, Intravenous ; Animals ; Dependovirus ; Genetic Therapy ; Humans ; Primates
    Language English
    Publishing date 2018-03-01
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1016/j.ymthe.2018.02.009
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 5640: Show issues Location:
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    Jg. 1995 - 2021: Lesesall (2.OG)
    ab Jg. 2022: Lesesaal (EG)

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  6. Article ; Online: Nicholas Muzyczka, PhD [1947-2023].

    Berns, Kenneth I / Byrne, Barry J / Flotte, Terence R / Samulski, R Jude / Srivastava, Arun

    Human gene therapy

    2023  Volume 34, Issue 3-4, Page(s) 81–82

    Language English
    Publishing date 2023-02-16
    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.2023.29235.nmu
    Database MEDical Literature Analysis and Retrieval System OnLINE

    Full text online

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    Zs.A 2988: Show issues Location:
    Je nach Verfügbarkeit (siehe Angabe bei Bestand)
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  7. Article ; Online: Meeting Report: 2022 Muscular Dystrophy Association Summit on 'Safety and Challenges in Gene Transfer Therapy'.

    Lek, Angela / Atas, Evrim / Hesterlee, Sharon E / Byrne, Barry J / Bönnemann, Carsten G

    Journal of neuromuscular diseases

    2023  Volume 10, Issue 3, Page(s) 327–336

    Abstract: Muscular Dystrophy Association (MDA) has invested over $125M in the development of gene therapy for neuromuscular disorders (NMD) over the past 20 years. As a lead initiator of progress in this important field of medicine and to help ensure continued ... ...

    Abstract Muscular Dystrophy Association (MDA) has invested over $125M in the development of gene therapy for neuromuscular disorders (NMD) over the past 20 years. As a lead initiator of progress in this important field of medicine and to help ensure continued progress towards therapies for patients, MDA organized a dedicated summit in January 2022 to address emerging challenges in safely delivering adeno-associated virus (AAV) mediated gene therapies with a focus on their application in NMD. In this meeting, chaired by Carsten Bönnemann (NINDS, NIH) and Barry Byrne (University of Florida), academic and industry experts and stakeholders convened to openly discuss adverse events linked to clinical trials, as well as other challenges emerging in preclinical studies associated with difficulties in the translation of AAV-mediated gene therapies.
    MeSH term(s) Humans ; Muscular Dystrophies/genetics ; Muscular Dystrophies/therapy ; Neuromuscular Diseases/genetics ; Neuromuscular Diseases/therapy ; Genetic Therapy/adverse effects ; Dependovirus/genetics
    Language English
    Publishing date 2023-02-21
    Publishing country Netherlands
    Document type Journal Article
    ISSN 2214-3602
    ISSN (online) 2214-3602
    DOI 10.3233/JND-221639
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  8. Article ; Online: Nicholas Muzyczka, PhD.

    Berns, Kenneth I / Byrne, Barry J / Flotte, Terence R / Samulski, R Jude / Srivastava, Arun

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

    2023  Volume 31, Issue 3, Page(s) 607

    Language English
    Publishing date 2023-02-10
    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.2023.01.027
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Zs.A 5640: Show issues Location:
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  9. Article: Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy.

    Shoti, Jakob / Qing, Keyun / Keeler, Geoffrey D / Duan, Dongsheng / Byrne, Barry J / Srivastava, Arun

    Molecular therapy. Methods & clinical development

    2023  Volume 31, Page(s) 101147

    Abstract: The first generation of adeno-associated virus (AAV) vectors composed of the naturally occurring capsids and genomes, although effective in some instances, are unlikely to be optimal for gene therapy in humans. The use of the first generation of two ... ...

    Abstract The first generation of adeno-associated virus (AAV) vectors composed of the naturally occurring capsids and genomes, although effective in some instances, are unlikely to be optimal for gene therapy in humans. The use of the first generation of two different AAV serotype vectors (AAV9 and AAVrh74) in four separate clinical trials failed to be effective in patients with Duchenne muscular dystrophy, although some efficacy was observed in a subset of patients with AAVrh74 vectors leading to US Food and Drug Administration approval (Elevidys). In two trials with the first generation of AAV9 vectors, several serious adverse events were observed, including the death of a patient in one trial, and more recently, in the death of a second patient in an N-of-1 clinical trial. In a fourth trial with the first generation of AAVrh74 vectors, myositis and myocarditis were also observed. Here, we report that capsid- and genome-modified optimized AAVrh74 vectors are significantly more efficient in transducing primary human skeletal muscle cells
    Language English
    Publishing date 2023-11-02
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2872938-9
    ISSN 2329-0501 ; 2329-0501
    ISSN (online) 2329-0501
    ISSN 2329-0501
    DOI 10.1016/j.omtm.2023.101147
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  10. Article ; Online: Considerations for Systemic Use of Gene Therapy.

    Byrne, Barry J / Corti, Manuela / Muntoni, Francesco

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

    2021  Volume 29, Issue 2, Page(s) 422–423

    MeSH term(s) Genetic Diseases, Inborn/genetics ; Genetic Diseases, Inborn/therapy ; Genetic Therapy/methods ; Humans
    Language English
    Publishing date 2021-01-14
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1016/j.ymthe.2021.01.016
    Database MEDical Literature Analysis and Retrieval System OnLINE

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