Leah Byrne, PhD

  • Assistant Professor of Ophthalmology
  • University of Pittsburgh School of Medicine
  • Gene Therapy for Retinal Disease Laboratory

Dr. Leah Byrne holds the position of Assistant Professor in the Department of Ophthalmology, with secondary appointments in the Departments of Neurobiology and Bioengineering. The Byrne Lab develops gene-based approaches, including viral vector-mediated gene delivery and genome editing, to interrogate the biology underlying retinal disease and treat inherited blindness.

Dr. Byrne attended Hamilton College, Clinton, New York, where she received a BA in Neuroscience. Dr. Byrne was a J. William Fulbright Fellow at Karolinksa Institutet in Stockholm, Sweden. She then attended the University of California Berkeley, Berkeley, California, where she received a PhD in Neuroscience.

Prior to working at Pitt, Dr. Byrne was a Ruth L. Kirschstein NRSA Postdoctoral Fellow and Ford Foundation Fellow at the University of California Berkeley and the University of Pennsylvania. During this time, she engineered next-generation AAV viruses for gene therapy in the retina, developed high throughput methods for directed evolution of viral vectors, and created therapies for inherited retinal degenerations affecting photoreceptors and RPE.

Dr. Byrne has received a Research to Prevent Blindness Career Development Award, a Foundation Fighting Blindness Individual Investigator Award, and the 2023 University of Pittsburgh Office of Innovation Emerging Innovator Award. Dr. Byrne has served as an Associate Scientific Advisor for Science Translational Medicine, and was named an Emerging Vision Scientist, National Alliance for Eye and Vision Research. Dr. Byrne holds patents on viral vectors and methods for the development of viral vectors for gene therapy. Dr. Byrne is also the co-founder and chief scientific officer of Avista Therapeutics, a company working to develop gene therapies for retinal degeneration.

Representative Publications

Dr. Byrne’s Publications

A list of publications can be found here: https://www.ncbi.nlm.nih.gov/myncbi/leah.byrne.1/bibliography/public/?so...

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 is developing new therapies to address these issues and restore vision, including genome editing and optogenetics approaches. The Byrne lab also 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.

Announcements
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: Research in the Byrne lab is supported by funding from the NIH, The FNIH, the Foundation Fighting Blindness, Research to Prevent Blindness, and UPMC Enterprises.