Translational Retinal Research and Therapies

Alison J Hardcastle; Paul A Sieving; José-Alain Sahel; Samuel G Jacobson; Artur V Cideciyan; John G Flannery; William A Beltran; Gustavo D Aguirre

doi:10.1167/tvst.7.5.8

Translational Retinal Research and Therapies

Transl Vis Sci Technol. 2018 Sep 13;7(5):8. doi: 10.1167/tvst.7.5.8. eCollection 2018 Sep.

Authors

Alison J Hardcastle¹, Paul A Sieving², José-Alain Sahel³, Samuel G Jacobson⁴, Artur V Cideciyan⁴, John G Flannery⁵, William A Beltran⁶, Gustavo D Aguirre⁶

Affiliations

¹ Professor of Molecular Genetics, UCL Institute of Ophthalmology, London, UK.
² Director, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
³ Department of Ophthalmology, University of Pittsburgh School of Medicine, Director of the UPMC Eye Center, University of Pittsburgh Medical Center, Pittsburgh, PA, USA and Director, Institut de la Vision, Sorbonne Université-Inserm-CNRS, Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, Paris, France.
⁴ Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
⁵ Department of Molecular and Cell Biology, Helen Wills Neuroscience Institute, University of California, Berkeley, CA, USA.
⁶ Department of Clinical Sciences and Advanced Medicine, Division of Experimental Retinal Therapies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.

Abstract

The following review summarizes the state of the art in representative aspects of gene therapy/translational medicine and evolves from a symposium held at the School of Veterinary Medicine, University of Pennsylvania on November 16, 2017 honoring Dr. Gustavo Aguirre, recipient of ARVO's 2017 Proctor Medal. Focusing on the retina, speakers highlighted current work on moving therapies for inherited retinal degenerative diseases from the laboratory bench to the clinic.

Keywords: Leber congenital amaurosis; X-linked Retinoschisis; animal models; retinitis pigmentosa.

Abstract

Grants and funding