Developing Gene Therapies for AMD: Brandi L. Williams, PhD; Gregory S. Hageman, PhD

Directors from the Moran Eye Center discussed researching gene therapy approaches targeting HTRA1 in AMD.

“It's important to keep an open mind for all research. A lot of times what's in the literature is based on model systems or other more contrived approaches. I think it's always worthwhile, if you have a fresh approach, to take it and see where it leads. We could have just accepted that what was in the literature was true.”

Multiple studies are currently investigating HTRA1 as a therapeutic target for downregulation to mitigate the development of age-related macular degeneration (AMD). Amid this avenue of research, researchers from the John A. Moran Eye Center at the University of Utah have discovered a contradictory protective effect of the gene on AMD development.

GeneTherapyLive spoke with Brandi. L Williams, PhD, research director, and Gregory S. Hageman, PhD, executive director, Steele Center for Translational Medicine, both from the John A. Moran Eye Center, to learn more about the importance of continuing research even in areas with established knowledge. 

Williams BL, Seager NA, Gardiner JD, et al. Chromosome 10q26–driven age-related macular degeneration is associated with reduced levels of HTRA1 in human retinal pigment epithelium. PNAS. 2021;118(30) e2103617118. doi:10.1073/pnas.2103617118
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