Rachel A. Silverstein

OptiPrime predicts prime editing outcomes with high accuracy, enabling efficient therapeutic applications in human and mouse cells.

This paper develops a customized CRISPR-Cas9 base editor to correct the ACTA2 R179H mutation in a mouse model of MSMDS, improving survival and rescuing vascular and neurological phenotypes.

This paper introduces Protein2PAM, a deep learning model that predicts CRISPR-Cas PAM specificity from protein sequences, enabling the design of Cas variants with tailored PAM recognition.

This study creates a variant effect map for GDI1 missense variants using functional assays and machine learning to distinguish pathogenic from benign variants.