Article

Tunable, proteolytic dosage control of CRISPR-Cas systems enables precise gene therapy for dosage sensitive disorders

2024-10-09

Abstract excerpt

<h4>Summary</h4> The ability to modulate gene expression through modular and universal genetic tools like CRISPR-Cas has greatly advanced gene therapy for therapeutics and basic science. Yet, the inherent stochasticity of delivery methods cause variation in target gene expression at the single-cell level, limiting their applicability in systems that require more precise expression. Thus, we implement a modular in...

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Literature Corpus work: a66a331d-2ae8-5533-a81d-b4fd96497a40
DOI: 10.1101/2024.10.09.617463

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