ArticleMolecular therapy : the journal of the American Society of Gene Therapy2022
AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
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Who cites it
27 citing papers in PubMed.
- RNA splicing and cardiovascular disease: a guide for cardiologists.European heart journal · 2026Review
- AAV Vector Toolkit for the Delivery and Expression of the Artificial microRNA in the Murine Heart.Biotech (Basel (Switzerland)) · 2026Article
- Adeno-Associated Virus Vector Mediated Gene Therapy: A Promising Approach to Transform Hypertrophic Cardiomyopathy Treatment.Biotechnology journal · 2026Review
- CircRNA-regulated programmed cell death networks in cardiomyocytes: Molecular crosstalk and therapeutic translation.Non-coding RNA research · 2026Review
- Histone Modifications in Cardiovascular Disease: Mechanisms and Therapeutic Opportunities.MedComm · 2026Review
- Long non-coding RNAs as molecular hubs integrating inflammatory and osteogenic pathways in calcific aortic valve disease.Frontiers in cardiovascular medicine · 2026Review
- Design, development, and evaluation of gene therapeutics specific to KSHV-associated diseases.Molecular therapy. Oncology · 2025Article
- Long Non-coding RNA Based Therapy for Cardiovascular Disease.Journal of cardiovascular translational research · 2025Review
- Deletion of METTL14, a key methylation regulator, attenuates vascular ageing.European heart journal · 2025Article
- Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates.Gene therapy · 2025Article
- AAV6 vectors provide superior gene transfer compared to AAV9 vectors following intramyocardial administration.Molecular therapy. Methods & clinical development · 2025Article
- Advances and Challenges in Adeno-Associated Virus Gene Therapy Applications of Localized Delivery Strategies.Current medical science · 2025Review
- Review
- Genetic Animal Models of Cardiovascular Pathologies.Biomedicines · 2025Review
- Profiling and functional characterization of long noncoding RNAs during human tooth development.International journal of oral science · 2025Article
- A roadmap toward genome-wide CRISPR screening throughout the organism.Cell genomics · 2025Review
- Emerging Roles of Long Non-Coding RNAs in Cardiovascular Diseases.Journal of cellular and molecular medicine · 2025Review
- The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2.Gene therapy · 2024Article
- Adeno-associated virus-based gene therapy treats inflammatory kidney disease in mice.The Journal of clinical investigation · 2024Article
- Therapeutic Application and Structural Features of Adeno-Associated Virus Vector.Current issues in molecular biology · 2024Review
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Authors and funding
14 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
AAV vectors are promising delivery tools for human gene therapy. However, broad tissue tropism and pre-existing immunity against natural serotypes limit their clinical use. We identified two AAV capsid variants, AAV2-THGTPAD and AAV2-NLPGSGD, by in vivo AAV2 peptide display library screening in a murine model of pressure overload-induced cardiac hypertrophy. Both variants showed significantly improved efficacy in in vivo cardiomyocyte transduction compared with the parental serotype AAV2 as indicated by a higher number of AAV vector episomes in the nucleus and significant improved transduction efficiency. Both variants also outcompeted the reference serotype AAV9 regarding cardiomyocyte tropism, reaching comparable cardiac transduction efficiencies accompanied with liver de-targeting and decreased transduction efficiency of non-cardiac cells. Capsid modification influenced immunogenicity as sera of mice treated with AAV2-THGTPAD and AAV2-NLPGSGD demonstrated a poor neutralization capacity for the parental serotype and the novel variants. In a therapeutic setting, using the long non-coding RNA H19 in low vector dose conditions, novel AAV variants mediated superior anti-hypertrophic effects and revealed a further improved target-to-noise ratio, i.e., cardiomyocyte tropism. In conclusion, AAV2-THGTPAD and AAV2-NLPGSGD are promising novel tools for cardiac-directed gene therapy outperforming AAV9 regarding the specificity and therapeutic efficiency of in vivo cardiomyocyte transduction.
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