Ruorong Yan, Valentina Cigliola, Kelsey A Oonk, Zachary Petrover, Sophia DeLuca, David W Wolfson, Andrew Vekstein, Michelle A Mendiola, Garth Devlin, Muath Bishawi, Matthew P Gemberling, Tanvi Sinha, Michelle A Sargent, Allen J York, Avraham Shakked, Paige DeBenedittis, David C Wendell, Jianhong Ou, Junsu Kang, Joseph A Goldman, Gurpreet S Baht, Ravi Karra, Adam R Williams, Dawn E Bowles, Aravind Asokan, Eldad Tzahor, Charles A Gersbach, Jeffery D Molkentin, Nenad Bursac, Brian L Black, Kenneth D Poss
The efficacy and safety of gene-therapy strategies for indications like tissue damage hinge on precision; yet, current methods afford little spatial or temporal control of payload delivery. Here, we find that tissue-regeneration enhancer elements (TREEs) isolated from zebrafish can direct targeted, injury-associated gene expression from viral DNA vectors delivered systemically in small and large adult mammalian species. When employed in combination with CRISPR-based epigenome editing tools in mice, zebrafish TREEs stimulated or repressed the expression of endogenous genes after ischemic myocardial infarction...
December 6, 2022: Cell Stem Cell