Article

Large-scale discovery of potent, compact and lineage specific enhancers for gene therapy vectors

2023-10-06

Abstract excerpt

Regulation of gene expression during cell development and differentiation is chiefly orchestrated by distal noncoding regulatory elements that precisely modulate cell selective gene activity. Gene therapy vectors rely on the cellular and context specificity of regulatory DNA elements to express therapeutic transgenes in the correct location and time. Here, we develop a straight-forward, one-shot approach to screen...

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Literature Corpus work: c6139036-21c8-593f-af37-84c19e2d454e
DOI: 10.1101/2023.10.04.559165

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