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Top stories on cardiac gene therapy delivery

Year: 2026

Type: article

Abstract: Gene therapy allows for the treatment of genetic diseases by targeting the underlying molecular cause. It necessitates delivering exogenous genetic material to affected tissue to silence, replace, or activate the expression of specific genes. Currently, viral vectors are the preferred method for achieving efficient delivery of genetic material. Among them, adeno-associated viruses (AAVs) are the leading platform in gene therapy owing to their favorable safety profile, efficacy, and versatility. In particular, AAV9 exhibits the strongest cardiac tropism, making it the vector of choice for gene therapy targeting heart disease.

Source: Heart Rhythm

DOI: 10.1016/j.hrthm.2025.10.041

Authors: Rescell M. Bianan, Yigal M. Pinto, Esther E. Creemers

Institutions: Amsterdam University Medical Centers, Amsterdam Cardiovascular Sciences, University of Amsterdam

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $4,790