We can’t show the full text here under this license.
Efficient RNA base editing in living cells using engineered enzymes combining cytidine deaminase and RNA-binding proteins
Updated
Abstract
Essence
Engineered APOBEC-PUF editors enabled C-to-U RNA editing with disease-relevant effects in mouse models.
Evidence
Preclinical RNA-editing platform experiments showed ProAPOBEC-PUF editing of Pcsk9 lowered cholesterol in mice and AAV brain editing corrected Mef2c mRNA mutations with phenotype improvement in an autism mouse model.
Caveat
The evidence is limited to mouse models, so human safety, delivery durability, and clinical efficacy remain untested.
Simplified
Key numbers
82.3%
Editing Efficiency Increase
Success rate of CU-REWIRE4.0 vs. CU-REWIRE3.0.
over 70%
Cholesterol Reduction
Reduction in cholesterol levels post -CU5.21 administration.
43%
Social Interaction Improvement
Editing efficiency on Mef2c mRNA in L35P mice.
CRISPR Gene Editing