• Leah Byrne, PhD

    Assistant Professor, Ophthalmology, University of Pittsburgh

    Research: “A modular system for gene and protein delivery to the retina based on AAV”


    “A modular system for gene and protein delivery to the retina based on AAV”

    The Byrne Lab develops gene therapies for retinal disease. Inherited retinal dystrophies include a diverse group of blinding disorders that have a profound impact on the quality of life of patients. Approximately 1 in 3000 people worldwide are affected by inherited retinal degenerations. This group of diseases involve mutations in more than 200 genes, with autosomal recessive, dominant, X-linked, and complex patterns of inheritance. There are currently no effective treatments for most forms of inherited retinal degeneration. However, gene therapy, in which a healthy copy of a mutated gene or a therapeutic protein is delivered to cells in the retina, is a highly promising approach to treating retinal disease. Before gene therapy strategies are effective, efficient and applicable to most retinal diseases, there are significant obstacles that must be overcome. These include developing gene therapy approaches for diseases involving large genes, dominant mutations, and mutations in non-coding regions. The Byrne lab uses high throughput methods, guided by computational approaches, to engineer viral vectors with new abilities and improved capabilities to deliver therapeutic genes to the retina. We are developing new therapies that allow for increased precision of gene delivery and protein expression. Additionally, a main focus of the lab is to develop and implement gene editing approaches using CRISPR/Cas9, a powerful and widely applicable molecular tool, which we are using to directly rewrite the genome.



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