Delivered by Dr. Edwin Stone at the International Symposium on Usher Syndrome.

Download the presentation slides here.

Stone, E.

University of Iowa, Iowa City, IA, USA

Abstract:

This talk will cover the following topics:
1) The inherited eye disease landscape
a) the treatments that will be useful for a given patient depend in part on that patient’s stage of disease — we should have a strategy that "leaves no one behind" regardless of their stage of their disease
b) the strategy for developing and deploying treatments depends to some degree on whether the disease is above or below the "commercial viability threshold”
2) Tiered genetic testing for genetically heterogeneous diseases — our current strategy and results
3) Strategic choices for gene therapy — including genome editing (TALEN and CRISPR) for correction of disease-causing mutations in vivo and in vitro
4) Strategic choices for cell-based therapy — including: patient derived stem cells as a means to:
a) investigate pathogenic mechanisms
b) evaluate therapies designed to prevent, arrest or slow the disease
c) replace photoreceptors to restore vision