Despite the significant advances made in recent years, there are still a number of obstacles standing in the way of wider application of gene therapies. These include the efficient delivery of genetic material to target cells with minimal side effects using adeno-associated viral (AAV) vectors. AAV carriers have an advantageous safety profile and high gene transfer efficiency, which means they are often used in gene therapy and CRISPR/Cas gene editing. But AAVs have limited DNA uptake capacity and cannot reliably transport larger genes. A team of researchers has developed a new approach to overcome these drawbacks. This new method, dubbed REVeRT (reconstitution via mRNA trans-splicing), also uses the principle of dual AAV vectors. However, unlike previous technologies, it relies on the assembly of gene fragments divided at the transcriptional level. The team has already developed the method for ophthalmological applications in cell cultures, and has successfully evaluated it in animal models under a variety of conditions, for example to treat hereditary macular degeneration by gene therapy.
