AAV vectors advance cardiac gene therapy while overcoming challenges.

The idea of gene therapy, in which nucleic acids are introduced into cells to restore lost functions, was proposed in the 1970s with the progress of molecular biology [1]. Development has focused on methods to replenish genes by expressing normal cDNAs of damaged genes in cells, but there are also methods to express miRNAs and shRNAs that suppress the expression of harmful genes. Recently, genome editing technologies have also started to be applied clinically. In the last decade, gene therapy developments have increased exponentially, with more than 2000 in the development pipeline, including targeting cardiovascular and muscular diseases [2].
Source: International Journal of Cardiology - Category: Cardiology Authors: Tags: Editorial Source Type: research