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A universal strategy for AAV delivery of base editors to correct genetic point mutations in neonatal PKU mice
A universal method using viral delivery to fix genetic mutations in newborn mice with PKU
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Abstract
Up to 27.7% correction of a phenylketonuria mutation was achieved using a dual adeno-associated virus strategy.
- Base editing tools can convert C:G or A:T base pairs to T:A or G:C, targeting specific genetic lesions.
- The efficiency of correcting disease-causing mutations has been limited by the size of existing base editors.
- A novel dual AAV strategy was designed to deliver base editors, linking deaminases to Cas9 using a specific peptide-antibody interaction.
- One or two copies of the GCN4 peptide were sufficient for assembling base editors, resulting in effective targeted editing.
- The dual AAV system successfully corrected PKU-related mutations in neonatal mice, alleviating symptoms of hyperphenylalaninemia.
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