Regenerative Gene Therapy for Heart Attack Harnesses Zebrafish TREE Elements
A team of Duke scientists studying how animals such as lizards and fish regrow damaged tissues has demonstrated the ability to control gene activity in response to injury, limiting it to a specific region of tissue and during a defined time window, rather than being continuously active in the entire organ. Their gene therapy approach harnessed zebrafish tissue-regeneration enhancer elements (TREEs), delivered systemically using adeno-associated viral (AAV) vectors to direct the expression of genes in a mouse model of heart injury, and improve cardiac function.
The Duke team, headed by said Ken Poss, PhD, the James B. Duke Distinguished Professor of Regenerative Biology in the Duke School of Medicine, reported on their developments in Cell Stem Cell, in a paper titled “An enhancer-based gene-therapy strategy for spatiotemporal control of cargoes during tissue repair.” In their paper, the team concluded, “Importantly, TREEs displayed specificity and efficacy when used as gene-therapy modules in systemically delivered recombinant AAV vectors in mice, including the capacity to localize cardiogenic events and improve cardiac function … Our study provides a foundation for new gene-therapy approaches to improve tissue repair in disease settings.”
If humans are ever going to be able to regrow damaged tissues the way lizards and fish routinely do, it will require the precise control of gene expression in time and place—to prevent random cells growing everywhere or creating a new body part that never quits growing.
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