Leah Byrne, PhD

  • Assistant Professor of Ophthalmology
  • University of Pittsburgh School of Medicine

Gene Therapy for Retinal Disease Laboratory

Representative Publications

Research Interest Summary

Gene therapy, viral vector engineering, genome editing, inherited retinal degeneration, genetics of eye disease

Research Interests

Lab Personnel

Oliver Beale, Medical Student, University of Pittsburgh School of Medicine

Esin Öztürk, PhD, Postdoctoral Associate, Ophthalmology

Sara Jabalameli, PSM, Research Specialist, Ophthalmology

Molly Johnson, Bioinformatics Specialist, Ophthalmology

Serhan Turunc, Research Fellow, Ophthalmology

Zhuahuan Xi, Medical Student and Visiting Scholar, Ophthlamology

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.

The Byrne lab is currently recruiting highly qualified applicants for a Postdoctoral Position in the area of AAV vector development. For more information, please contact Dr. Byrne.

Research Grants

Funding Support
UPP Academic Foundation

Research to Prevent Blindness

Usher1F Collaborative

National Eye Institute

The Foundation Fighting Blindness

E. Matilda Ziegler Foundation