Targeting the liver to treat the eye

• Published in: EMBO molecular medicine Vol. 15; no. 4; pp. e17285 - n/a
• Main Authors: Seker Yilmaz, Berna, Gissen, Paul
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 11.04.2023; EMBO Press; John Wiley and Sons Inc; Springer Nature
• Subjects: Age; Biomedicine; Dependovirus - genetics; Disease; EMBO16; Enzymes; Expression vectors; FDA approval; Gene therapy; Genetic Therapy; Genetic Vectors; Genome editing; Hemophilia; Humans; Liver; Metabolism; Molecular Medicine; News & Views; Ornithine-aminotransferase; Plasma; Proteins; Serotypes; Views
• DOI: 10.15252/emmm.202217285
• ISSN: 1757-4676

Over the last two decades, gene therapy has given hope of potential cure for many rare diseases. In the simplest form, gene therapy is the transfer or editing of a genetic material to cure a disease via nonviral or viral vehicles. Gene therapy can be performed either in vivo by injecting a vector carrying the gene or tools for gene editing directly into a tissue or into the systemic circulation, or ex vivo when patient cells are genetically modified outside of the body and then introduced back into the patient (Yilmaz et al , 2022). Adeno‐associated viral vectors (AAV) have been the vectors of choice for in vivo gene therapy. There has been a lot of promising research on the development of novel tissue and cell‐specific serotypes in order to improve efficacy and safety for clinical applications (Kuzmin et al , 2021). In this issue of EMBO Molecular Medicine, Boffa and colleagues present a novel AAV‐based liver‐directed gene therapy for ornithine aminotransferase deficiency. Graphical Abstract P Gissen and B Şeker Yilmaz discuss novel liver‐directed gene therapy for gyrate atrophy of the choroid and retina due to ornithine aminotransferase deficiency as reported by N Brunetti‐Pierri and colleagues, in this issue of EMBO Mol Med .