Seker Yilmaz, Berna;
Gissen, Paul;
(2023)
Targeting the liver to treat the eye.
EMBO Molecular Medicine
, Article e17285. 10.15252/emmm.202217285.
Preview |
Text
Targeting the liver to treat the eye.pdf - Published Version Download (670kB) | Preview |
Abstract
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.
Type: | Article |
---|---|
Title: | Targeting the liver to treat the eye |
Location: | England |
Open access status: | An open access version is available from UCL Discovery |
DOI: | 10.15252/emmm.202217285 |
Publisher version: | https://doi.org/10.15252/emmm.202217285 |
Language: | English |
Additional information: | © 2023 The Authors. Published under the terms of the CC BY 4.0 license This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited. |
UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Genetics and Genomic Medicine Dept |
URI: | https://discovery.ucl.ac.uk/id/eprint/10166024 |
Archive Staff Only
![]() |
View Item |