Blind Children’s Center Awards Grant for Gene Therapy Research to Combat CEP290-LCA
For more than 25 years, the Blind Children’s Center’s Lisa D. Hansen Medical Research Grant Program has supported scientific research seeking cures for juvenile blindness. Lisa D. Hansen—a longtime board member, donor and dear friend to the BCC community—was a champion of the research grant program through which BCC awards annual grants to scientists who pursue preventing visual impairments in very young children.
“The purpose of our research grant is to support the prevention of vision loss in young children,” said Research Committee Chair Sherwin J. Isenberg, M.D.. “By investing in innovative research, the BCC can unlock new possibilities and bring hope to children and families facing the challenges of visual impairments.”
The Research Committee, composed of 10 education and medical professionals, has awarded the BCC’s 2024 research grant to Seongjin Seo, Ph.D., Principal Investigator at the Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa Carver College of Medicine. The focus of Dr. Seo’s research is the development of mutation-independent, generic gene therapy vectors to treat CEP290-associated Leber Congenital Amaurosis (CEP290-LCA).
Leber Congenital Amaurosis (LCA) is a rare inherited eye disease that appears at birth or in the first few months of life, characterized by severe vision loss or blindness. Among the various genetic mutations that can cause LCA, those affecting the CEP290 gene are particularly significant, making this research crucial for advancing treatment options. The proposed study aims to evaluate and compare the therapeutic efficacy of two sets of AAV-CEP290 vectors in animal models of CEP290-LCA, which will not only advance the development of effective mutation-independent gene therapy vectors, but also expand the potential of gene therapy in treating other inherited retinal diseases.
“Receiving this grant from the BCC provides a crucial opportunity to push the boundaries of gene therapy,” shared Dr. Seo. “With this support, we are one step closer to developing effective treatments that could transform the future for children with visual impairments.”
By supporting innovative studies, BCC continues to lead efforts in transforming scientific discoveries into tangible solutions that enhance the lives of children affected by visual impairments. For more information about BCC’s Research Committee and previous awardees, visit: www.blindchildrenscenter.org/medical-research/.
