Top stories on cardiac gene therapy delivery

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.