Gerald Schwank

This paper uses prime editing to classify LDLR variants by measuring their impact on LDL-C uptake, reclassifying many previously unclassified variants.

The paper improves the activity of compact RNA-guided nucleases in mammalian cells through directed evolution, resulting in variants with higher editing efficiency and reduced off-target effects.

The paper develops CRISPR-PAMdb and CICERO to uncover PAM diversity in Cas9 proteins, enabling better CRISPR tool design.

The paper develops scOUT-seq to profile DNA repair outcomes in single cells and finds that repair outcomes vary significantly across cell types and subtypes.

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.

Hallucinating binders by backpropagating through AlphaFold2 weights gave, across 12 targets, binders in 10-100% of the designs tested per target, mostly at nanomolar affinity.

This paper describes a gene therapy approach using liver cell transplantation to treat PKU in mice by modifying hepatocytes to resist paracetamol and replace diseased cells.

This study uses prime editing in mice brains to introduce a mutation in the Adrb1 gene, leading to increased activity and exploratory behavior.

The paper describes a minimally invasive method for delivering AAV vectors to pig livers but finds low vector retention and no detectable editing in vivo.

This paper develops BE-DICT, a deep learning model trained on high-throughput screens to predict base editing outcomes with high accuracy.

In vivo prime editing corrects phenylketonuria in mice by repairing the Pah gene mutation and reducing blood phenylalanine levels.

This study shows that covalently linking a DNA repair template to the CRISPR/Cas9 complex increases HDR efficiency 23-fold compared to NHEJ.