Neurosensory Disorders

Neurosensory disorders such as vision and hearing loss are debilitating disorders that can dramatically affect quality of life, often with few treatment options available for patients. Gene therapy is a promising modality to intervene in the disease process that lead to disorders of the senses. Its most immediate application has been in relatively rare inherited forms of retinal degeneration such as Retinitis Pigmentosa and Leber Congenital Amaurosis. These patient lose visual function throughout life due to mutations in a single gene, and gene therapy approaches aim at delivering a gene that mitigates the disease causing effects of this one mutated gene.

This area of gene therapy has clinically progressed quite far and may soon lead to drug approval for selected blinding disorders with many others following at various stages of development. The progress in this field has led others to also target disorders that are more common but also more complex in that multiple factors, genetic and environmental, contribute to the disease process such as Age-Related Macular Degeneration and hearing loss. 


22nd Annual Meeting
April 29 – May 2 | Washington D.C.