Péter István Kulcsár

This paper demonstrates transient prime editor expression in mice to correct a genetic liver disease, reducing blood phenylalanine levels below the therapeutic threshold.

This study uses base editing to downregulate PTBP1 in neurons of a PD mouse model, rescuing motor symptoms without affecting astrocytes.

This study compares SuperFi-Cas9 to first-generation IFN variants, finding it has high fidelity but reduced activity, and shows improved specificity when paired with ABE8e.

The paper develops a set of high-fidelity SpCas9 variants that enable efficient and specific genome editing across all target sites without off-target effects.