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Peer-reviewedCC BY 4.0Open Heart

Evaluating selected gene therapies for cardiovascular diseases: an updated scoping review and evidence map of randomised controlled trials

  • Eric A Apaydin1,2,3
  • Jessica Richard4
  • Skye A Miner5
  • Molly Waymouth1
  • Derek Roberts1
  • Jerry Vockley6
  • Andrea S Richardson7
7 affiliations
  1. 1RAND Health, RAND Corporation, Santa Monica, California, USA
  2. 2Department of Medicine, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, California, USA
  3. 3Center for Healthcare Innovation, Implementation, and Policy, VA Greater Los Angeles Heatlhcare System, Los Angeles, California, USA
  4. 4RAND Health, RAND Corporation, Arlington, Virginia, USA
  5. 5RAND Health, RAND Corporation, Boston, Massachusetts, USA
  6. 6Department of Pediatrics, School of Medicine, University of Pittsburgh, Pittsburgh, Pennsylvania, USA
  7. 7RAND Health, RAND Corporation, Pittsburgh, Pennsylvania, USA
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Abstract

Objective: Gene therapies are increasingly being developed and evaluated to treat complex diseases with multiple genetic causes, including cardiovascular diseases (CVDs). However, these studies have not yet been reviewed systematically. We aimed to conduct a scoping review of randomised controlled trials of gene therapies for CVDs and create an evidence map of the included literature.

Methods: We searched six databases for studies published from November 2018 to March 2025. Our search yielded 9106 records, and 1864 publications were reviewed at the full-text level.

Results: We included 28 randomised controlled trials (RCTs) evaluating interventions delivered by RNA interference (13 RCTs; 46%), antisense oligonucleotides (9 RCTs; 32%), plasmids (4 RCTs; 14%) and other vectors (adeno-associated virus and adenovirus; 2 RCTs; 7%). Cardiovascular indications included dyslipidaemia (12 RCTs; 43%), ischaemia (4 RCTs; 14%), transthyretin amyloidosis (4 RCTs; 14%), heart failure (2 RCTs; 7%) and other CVDs (6 RCTs; 21%). Samples included an average of 183 participants (range: 5–655); studies followed these participants for an average of 13 months (range: 3–97). Interventions were efficacious in most (22 RCTs; 85%) of the 26 studies that reported efficacy outcomes, but effects lasted less than 1 year in most studies (69%; 18 RCTs). Serious adverse events varied widely by study (range: 0%–62% of intervention group participants), but 19 RCTs reported that less than 10% of intervention group participants experienced a SAE.

Conclusions: Gene therapies for CVDs were reported to be efficacious in most RCTs, but their effects lasted less than 1 year in 69% of studies. Gene therapies for CVDs show promise in their efficacy and relative safety, but more research is needed to evaluate their long-term effects and risks.

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Cite

BibTeXRIS
BibTeX
@article{apaydin2026evaluating,
  title = {{Evaluating selected gene therapies for cardiovascular diseases: an updated scoping review and evidence map of randomised controlled trials}},
  author = {Eric A Apaydin and Jessica Richard and Skye A Miner and Molly Waymouth and Derek Roberts and Jerry Vockley and Andrea S Richardson},
  journal = {Open Heart},
  year = {2026},
  month = oct,
  doi = {10.1136/openhrt-2026-004374},
  url = {https://doi.org/10.1136/openhrt-2026-004374},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - Evaluating selected gene therapies for cardiovascular diseases: an updated scoping review and evidence map of randomised controlled trials
AU  - Eric A Apaydin
AU  - Jessica Richard
AU  - Skye A Miner
AU  - Molly Waymouth
AU  - Derek Roberts
AU  - Jerry Vockley
AU  - Andrea S Richardson
PY  - 2026
DA  - 2026/10/20
JO  - Open Heart
DO  - 10.1136/openhrt-2026-004374
UR  - https://doi.org/10.1136/openhrt-2026-004374
AB  - Objective: Gene therapies are increasingly being developed and evaluated to treat complex diseases with multiple genetic causes, including cardiovascular diseases (CVDs). However, these studies have not yet been reviewed systematically. We aimed to conduct a scoping review of randomised controlled trials of gene therapies for CVDs and create an evidence map of the included literature.  Methods: We searched six databases for studies published from November 2018 to March 2025. Our search yielded 9106 records, and 1864 publications were reviewed at the full-text level.  Results: We included 28 randomised controlled trials (RCTs) evaluating interventions delivered by RNA interference (13 RCTs; 46%), antisense oligonucleotides (9 RCTs; 32%), plasmids (4 RCTs; 14%) and other vectors (adeno-associated virus and adenovirus; 2 RCTs; 7%). Cardiovascular indications included dyslipidaemia (12 RCTs; 43%), ischaemia (4 RCTs; 14%), transthyretin amyloidosis (4 RCTs; 14%), heart failure (2 RCTs; 7%) and other CVDs (6 RCTs; 21%). Samples included an average of 183 participants (range: 5–655); studies followed these participants for an average of 13 months (range: 3–97). Interventions were efficacious in most (22 RCTs; 85%) of the 26 studies that reported efficacy outcomes, but effects lasted less than 1 year in most studies (69%; 18 RCTs). Serious adverse events varied widely by study (range: 0%–62% of intervention group participants), but 19 RCTs reported that less than 10% of intervention group participants experienced a SAE.  Conclusions: Gene therapies for CVDs were reported to be efficacious in most RCTs, but their effects lasted less than 1 year in 69% of studies. Gene therapies for CVDs show promise in their efficacy and relative safety, but more research is needed to evaluate their long-term effects and risks.
SN  - 2053-3624
ER  -