TY - JOUR
T1 - State of Gene Therapy for Monogenic Cardiovascular Diseases
AU - Bains, Sahej
AU - Giudicessi, John R.
AU - Odening, Katja E.
AU - Ackerman, Michael J.
N1 - Publisher Copyright:
© 2023 Mayo Foundation for Medical Education and Research
PY - 2024/4
Y1 - 2024/4
N2 - Over 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.
AB - Over 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.
UR - http://www.scopus.com/inward/record.url?scp=85188710296&partnerID=8YFLogxK
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U2 - 10.1016/j.mayocp.2023.11.003
DO - 10.1016/j.mayocp.2023.11.003
M3 - Review article
AN - SCOPUS:85188710296
SN - 0025-6196
VL - 99
SP - 610
EP - 629
JO - Mayo Clinic proceedings
JF - Mayo Clinic proceedings
IS - 4
ER -