Gene Therapy for Retinal Disease Laboratory

Principal Investigator

Leah Byrne, PhD

Assistant Professor of Ophthalmology
University of Pittsburgh School of Medicine


Lab Personnel

Oliver Beale 
Molly Johnson, Bioinformatics Specialis
Alessandra Larimer-Picciani
Bilge Esin Öztürk, PhD, Postdoctoral Fellow
Zhuahuan Xi, Medical Student and Visiting Scholar


Research Interests

  • Gene therapy
  • Viral vector engineering
  • Genome editing
  • Inherited retinal degeneration
  • Genetics of eye disease

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

UPP Academic Foundation

Research to Prvent Blindness

Usher1F Collaborative

National Eye Institute

The Foundaiton Fighting Blindness

E. Matilda Ziegler Foundation


Contact Information

Leah Byrne, PhD

3501 5th Ave.
Room 10044 BST3
Pittsburgh, PA 15213
The Byrne Lab