Leah C. Byrne
University of Pittsburgh
Publications 8
Delivery of a modified factor VIII gene leads to dramatic improvement of severe hemophilia A in human trials.
Transplantation of autologous transgenic stem cells resulted in near complete skin regeneration in a 7-year-old patient with severe junctional epidermolysis bullosa.
Based on observations of the mechanism underlying target discrimination, researchers have developed highly accurate variants of CRISPR-Cas9.
Researchers have edited the genome of human embryos to correct dominant myocardial disease.
Investigators from two teams have created personalized vaccines for the treatment of melanoma by identifying tumor-specific neoantigens.
A single intravitreal injection of AAV2 provides sustained delivery of anti-VEGF protein for the treatment of neovascular AMD.
Transplanted RPE cells derived from induced pluripotent stem cells maintained vision and were well tolerated in a patient with age-related macular degeneration.
A report of a patient treated with ex vivo lentiviral gene transfer to hematopoietic stem cells shows the promise of gene therapy for sickle cell anemia.