AUTHOR=Hanlon Killian S. , Chadderton Naomi , Palfi Arpad , Blanco Fernandez Alfonso , Humphries Peter , Kenna Paul F. , Millington-Ward Sophia , Farrar G. Jane TITLE=A Novel Retinal Ganglion Cell Promoter for Utility in AAV Vectors JOURNAL=Frontiers in Neuroscience VOLUME=11 YEAR=2017 URL=https://www.frontiersin.org/journals/neuroscience/articles/10.3389/fnins.2017.00521 DOI=10.3389/fnins.2017.00521 ISSN=1662-453X ABSTRACT=
Significant advances in gene therapy have enabled exploration of therapies for inherited retinal disorders, many of which are in preclinical development or clinical evaluation. Gene therapy for retinal conditions has led the way in this growing field. The loss of retinal ganglion cells (RGCs) is a hallmark of a number of retinal disorders. As the field matures innovations that aid in refining therapies and optimizing efficacy are in demand. Gene therapies under development for RGC-related disorders, when delivered with recombinant adeno associated vectors (AAV), have typically been expressed from ubiquitous promoter sequences. Here we describe how a novel promoter from the murine