Bioengineering Seminar Series - Xin "Daniel" Gao

Xin "Daniel" Gao, Assistant Professor, Department of Ophthalmology at the University of Pittsburgh, will present "Advancing Translational Gene Editing: From Targeted Correction to Scalable Gene Integration." Abstract In this talk, I will present two advances in therapeutic genome editing. First, I will describe an in vivo adenine base editing strategy that corrects the common Pex1 pathogenic allele in mouse models of Zellweger spectrum disorder, rescuing liver pathophysiology, peroxisomal function, and body weight. Second, I will discuss the development of evolved recombinases and prime editing — a platform enabling efficient, site-specific integration of large DNA sequences at defined genomic loci. Together, these approaches illustrate a progression from precise single-nucleotide correction to scalable, mutation-agnostic gene integration, advancing the translational potential of next-generation gene editing tools. Biography Daniel Gao is an Assistant Professor at the University of Pittsburgh Department of Ophthalmology. He is also a member of the Institute for Synthetic Biology, Center for Transcriptional Medicine at Pitt. His laboratory integrates biomolecular engineering, gene editing, and chemical biology to study genetic information, improve drug delivery, and enable the next generation of genomic medicine. During his PhD training at the University of Massachusetts Medical School, Dr. Gao developed an RNA-programmable proximity labeling platform that enables DNA-specific functional proteomics, providing new mechanistic insights into gene regulation and genome organization. During his postdoctoral training at Harvard University and the Broad Institute, Dr. Gao pioneered programmable gene integration and demonstrated therapeutic precision gene editing for Zellweger spectrum disorder and retinitis pigmentosa in preclinical mouse models. His work has resulted in nine granted patents and patent applications, several of which have been licensed by biopharmaceutic