Consideration of clinical translation of cardiac AAV gene therapy
Open accessAbstract
Advancements in conventional cardiac care have significantly reduced mortality from coronary heart disease and acute myocardial infarction. However, the prevalence of heart failure continues to increase in an aging population with profound social and economic consequences. Cardiac gene therapy with adeno-associated virus (AAV) vectors is emerging as a potential modality for addressing this desperate clinical need. After showing initial promise in extensive preclinical studies and an early clinical trial, disappointing results of large-scale clinical trial derailed the progress of AAV-mediated cardiac gene therapy. However, it appears that knowledge gained from previous failures coupled with developments in targeted gene delivery have set the stage for a new frontier in cardiac AAV gene therapy.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
The above data strongly indicated that optimal titers are critical for the success of cardiac gene therapies in humans, in addition to other considerations such as delivery method, vector serotype and tropism, and potential side effects.
“Without positive transduction, gene therapy cannot be effective; the above data strongly indicated that optimal titers are critical for the success of cardiac gene therapies in humans, in addition to other considerations such as delivery method, vector serotype and tropism, and potential side effects.78”
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
Heart disease and stroke statistics-2018 update: a report from the american heart association
Benjamin, E. J., Virani, S. S., Callaway, C. W., Chamberlain, A. M., Chang, A. R., Cheng, S., Chiuve, S. E., Cushman, M., Delling, F. N., Deo, R., et al. (2018) · Circulation · doi:10.1161/cir.0000000000000558 · Cited by 7,146 · see citing works
Greenberg, B., Butler, J., Felker, G. M., Ponikowski, P., Voors, A. A., Desai, A. S., Barnard, D., Bouchard, A., Jaski, B., Lyon, A. R., et al. (2016) · The Lancet · doi:10.1016/s0140-6736(16)00082-9 · Cited by 525 · see citing works
MicroRNA therapy stimulates uncontrolled cardiac repair after myocardial infarction in pigs
Gabisonia, K., Prosdocimo, G., Aquaro, G. D., Carlucci, L., Zentilin, L., Secco, I., Ali, H., Braga, L., Gorgodze, N., Bernini, F., et al. (2019) · Nature · doi:10.1038/s41586-019-1191-6 · Cited by 478 · see citing works
Recombinant Adeno-Associated Virus Serotype 9 Leads to Preferential Cardiac Transduction In Vivo
Pacak, C. A., Mah, C. S., Thattaliyath, B. D., Conlon, T. J., Lewis, M. A., Cloutier, D. E., Zolotukhin, I., Tarantal, A. F., & Byrne, B. J. (2006) · Circulation Research · doi:10.1161/01.res.0000237661.18885.f6 · Cited by 406 · see citing works
Cardiovascular Gene Therapy: Past, Present, and Future
Ylä‐Herttuala, S., & Baker, A. H. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.027 · Cited by 166 · see citing works
Gene therapy for the treatment of heart failure: promise postponed
Hulot, J.-S., Ishikawa, K., & Hajjar, R. J. (2016) · European Heart Journal · doi:10.1093/eurheartj/ehw019 · Cited by 150 · see citing works
Self-citationKawase, Y., & Hajjar, R. J. (2008) · Nature Clinical Practice Cardiovascular Medicine · doi:10.1038/ncpcardio1301 · Cited by 118 · see citing works
Vassalli, G., Büeler, H., Dudler, J., von Segesser, . K., & KAPPENBERGER, L. J. (2003) · International Journal of Cardiology · doi:10.1016/s0167-5273(02)00554-5 · Cited by 117 · see citing works
Hulot, J.-S., Salem, J.-E., Redheuil, A., Collet, J.-P., Varnous, S., Jourdain, P., Logeart, D., Gandjbakhch, E., Bernard, C., Hatem, S. N., et al. (2017) · European Journal of Heart Failure · doi:10.1002/ejhf.826 · Cited by 113 · see citing works
Gao, G., Bish, L. T., Sleeper, M. M., Mu, X. N., Sun, L. P., Lou, Y., Duan, J., Hu, C., Wang, L., & Sweeney, H. L. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.042 · Cited by 54 · see citing works
Rubanyi, G. M. (2013) · Molecular Therapy · doi:10.1038/mt.2013.13 · Cited by 44 · see citing works
Flagged first, then by citations
Research works printed but not linked: 1
- [5]Jessup M, Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID): a phase 2 trial of intracoronary gene therapy of sarcoplasmic reticulum Ca2+-ATPase in patients with advanced heart failure. Circulation 124, 304– 313 (2011). 21709064 PMC5843948
Cited by
Sorted by citations
Costa, K. D., Wong, A. O. T., Mak, S. Y., Roberts, E. G., Keung, W., Correia, C., Walentinsson, A., Christoffersson, J., Cheung, A., Lieu, D. K., et al. (2024) · bioRxiv · doi:10.1101/2024.08.27.609034 · Cited by 5 · see citing works
- Authors
- Yamada, K. P., Tharakan, S., & Ishikawa, K.
- Affiliations
- Icahn School of Medicine at Mount Sinai (US)
- Venue
- Cell and Gene Therapy Insights
- Publisher
- BioInsights Publishing, Ltd.
- Published
- 2020-05-14
- Volume
- 6
- Issue
- 5
- Pages
- 609-615
- ISSN
- 2059-7800 (ISSN-L)
- PMC
- PMC7406214
- OpenAlex
- W3027478874
- Cited by
- 13
- References
- 11
- Citations by year
- 2026: 2 · 2025: 4 · 2024: 2 · 2023: 1 · 2022: 3 · 2021: 1
- FWCI
- 0.73
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Cell and Gene Therapy Insights (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Heart, Lung, and Blood Institute (R01HL139963)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Cardiac electrophysiology and arrhythmias
- Keywords
- adeno-associated virus vectors, cardiac gene therapy, heart failure, targeted gene delivery, clinical translation, gene therapy clinical trials
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
