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THE EVOLVING ROLE OF MEDICAL GENETICISTS IN THE ERA OF GENE THERAPY: AN URGENCY TO PREPARE

  • Jerry Vockley
    Correspondence
    Correspondence: Dr. Jerry Vockley, University of Pittsburgh School of Medicine, UPMC Children's Hospital of Pittsburgh, 4401 Penn Avenue, Pittsburgh, PA 15224, (412) 692-7775,
    Affiliations
    University of Pittsburgh School of Medicine and UPMC Children’s Hospital of Pittsburgh, Pittsburgh, PA 15224, USA
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  • Author Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Nicola Brunetti-Pierri
    Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Affiliations
    Department of Translational Medicine, Federico II University, Naples, Italy

    Telethon Institute of Genetics and Medicine, Pozzuoli, Italy
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  • Author Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Wendy K. Chung
    Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Affiliations
    Division of Molecular Genetics, Departments of Pediatrics and Medicine, Columbia University, New York, NY, USA
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  • Author Footnotes
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    Angus J. Clarke
    Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Affiliations
    Cancer & Genetics, School of Medicine, Cardiff University, Wales, United Kingdom
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  • Author Footnotes
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    Nina Gold
    Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Affiliations
    Mass General Hospital for Children, Division of Medical Genetics and Metabolism and Harvard Medical School, Boston, Massachusetts, USA
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  • Author Footnotes
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    Robert C. Green
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    ∗ Contributed equally and are therefore listed alphabetically
    Affiliations
    MassGeneralBrigham, Ariadne Labs, Broad Institute and Harvard Medical School, Boston, MA, USA
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  • Author Footnotes
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    Stephen Kagan
    Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Affiliations
    Gene Therapy Platform and Cross Portfolio, Global Medical Affairs, Rare Disease, Pfizer Inc
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  • Author Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Tara Moroz
    Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Affiliations
    Gene Therapy Platform and Cross Portfolio, Global Medical Affairs, Rare Disease, Pfizer Inc
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  • Author Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Christian P. Schaaf
    Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Affiliations
    Institute of Human Genetics, Heidelberg University, Heidelberg, Germany
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  • Author Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Martin Schulz
    Footnotes
    ∗ Contributed equally and are therefore listed alphabetically
    Affiliations
    Gene Therapy Platform and Cross Portfolio, Global Medical Affairs, Rare Disease, Pfizer Inc
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  • Elfride De Baere
    Affiliations
    Center for Medical Genetics, Ghent University Hospital and Department of Biomolecular Medicine, Ghent University, C. Heymanslaan 10, B-9000 Ghent, Belgium
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    ∗ Contributed equally and are therefore listed alphabetically
Published:January 18, 2023DOI:https://doi.org/10.1016/j.gim.2023.100022
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      ABSTRACT

      By 2030, it is estimated that at least 30 non-oncology gene therapies will be approved in the United States alone. These therapies could be used to treat up to 50,000 patients annually and have the potential to result in major shifts in disease management pathways. Medical geneticists have well-established roles in the direct management of many rare genetic diseases and often provide support in the diagnosis and care of patients with such diseases. Because an increasing number of gene therapies are likely to become available over the next decade, there is a need to better define the role of medical geneticists within current and future gene therapy pathways and prepare for their expected role within the context of this new treatment paradigm. This commentary examines the current and potential future roles of medical geneticists in gene therapy and identifies specific needs that must be addressed for medical geneticists to assume an expanded leadership role in this area.

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