• Mayo Clinic proceedings · Apr 2024

    Review

    State of Gene Therapy for Monogenic Cardiovascular Diseases.

    • Sahej Bains, John R Giudicessi, Katja E Odening, and Michael J Ackerman.
    • Mayo Clinic Medical Scientist Training Program, Mayo Clinic Alix School of Medicine, Mayo Clinic, Rochester, MN; Department of Molecular Pharmacology and Experimental Therapeutics (Windland Smith Rice Sudden Death Genomics Laboratory), Mayo Clinic, Rochester, MN.
    • Mayo Clin. Proc. 2024 Apr 1; 99 (4): 610629610-629.

    AbstractOver the past 2 decades, significant efforts have been made to advance gene therapy into clinical practice. Although successful examples exist in other fields, gene therapy for the treatment of monogenic cardiovascular diseases lags behind. In this review, we (1) highlight a brief history of gene therapy, (2) distinguish between gene silencing, gene replacement, and gene editing technologies, (3) discuss vector modalities used in the field with a special focus on adeno-associated viruses, (4) provide examples of gene therapy approaches in cardiomyopathies, channelopathies, and familial hypercholesterolemia, and (5) present current challenges and limitations in the gene therapy field.Copyright © 2023 Mayo Foundation for Medical Education and Research. Published by Elsevier Inc. All rights reserved.

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