Tunable, proteolytic dosage control of CRISPR-Cas systems enables precise gene therapy for dosage sensitive disorders
Noa Katz, Connie An, Minza Haque, Sophia Mahiout, Yu‐Ju Lee, Josh Tycko, Meng Zhang, Jeewoo Kang, Lacramioara Bintu, Michael Cory Bassik, Wei‐Hsiang Huang, Xiaojing J. Gao
This paper introduces an IFFL-based CRISPR-Cas system to reduce gene expression variability, demonstrating improved dosage control for RAI1 in a mouse model.
bioRxiv · Oct 9, 2024 · PreprintSynthetic Biology