Large-scale discovery of potent, compact and lineage specific enhancers for gene therapy vectors
Nikoletta Psatha, Pavel Sova, Grigorios Georgolopoulos, Kiriaki Paschoudi, Mineo Iwata, Jordan E. Bloom, Tatyana Ulyanova, Hao Wang, Alexandra Kirtsou, Ninos-Ioannis Vasiloudis, Matthew S. Wilken, John A. Stamatoyannopoulos, Evangelia Yannaki, Thalia Papayannopoulou, George Stamatoyannopoulos, Jeff D. Vierstra
The paper identifies potent, compact, and lineage-specific enhancers for gene therapy vectors using epigenomic data and validates their efficacy in correcting a thalassemia phenotype.
bioRxiv · Oct 4, 2023 · PreprintGenomics