Article

Engineering a human-based translational activator for targeted protein expression restoration

2025-07-09

Abstract excerpt

Therapeutic modalities to programmably increase protein production are in critical need to address diseases caused by deficient gene expression via haploinsufficiency. Restoring physiological protein levels by increasing translation of their cognate mRNA would be an advantageous approach to correct gene expression, but has not been evaluated in an in vivo disease model. Here, we investigated if a translational ac...

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Literature Corpus work: 6465ef72-d273-5b98-8876-33716c241b4b
DOI: 10.1101/2025.07.09.663984

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