Markus Grompe

The paper develops an in vivo mouse model to assess the differential toxicity of cancer drugs on Fanconi Anemia mutant hematopoietic cells and identifies drugs with neutral toxicity.

The paper evaluates AAV capsids for liver gene therapy in a pig xenograft model, identifying efficient capsids for transgene expression in porcine hepatocytes.

This study evaluates the long-term combination therapy of metformin and oxymetholone in Fancd2-/- mice, finding no synergistic effect but improved HSC quiescence and partial gene expression normalization with metformin alone.

This paper corrects a mouse model of phenylketonuria by transplanting Cypor-deficient hepatocytes selected with acetaminophen, achieving stable normalization of blood phenylalanine levels.

The paper constructs a human DNA methylation atlas using whole-genome bisulfite sequencing of 39 cell types, revealing cell type-specific methylation patterns and regulatory elements.

This study shows FANCD2 reduces replication stress in fetal liver HSC, preventing excessive RPA-coated ssDNA and pChk1 activation.

The paper develops a barcoded AAV capsid library to identify novel gene therapy vectors with improved transduction efficiency in human islets and hepatocytes.

The paper develops a method to determine the tissue origins of circulating cell-free DNA using a methylation atlas and validates it in healthy and disease states.

The paper identifies methylation patterns specific to intestinal epithelial cells to detect cell-free DNA in plasma, showing increased levels in advanced CRC compared to IBD and PDAC.