CRISPR–Cas genome-editing nucleases hold substantial promise for developing human
therapeutic applications 1 , 2 , 3 , 4 , 5 – 6 but identifying unwanted off-target mutations is …

Tumors frequently display a glycolytic phenotype with increased flux through glycolysis and
concomitant synthesis of lactate. To maintain glycolytic flux and prevent intracellular …

While mechanisms of cytotoxicity and cytostaticity have been studied extensively from the
biological side, relatively little is currently understood regarding areas of chemical space …

Rheumatoid arthritis is a progressive, highly debilitating disease where early diagnosis,
enabling rapid clinical intervention, would provide obvious benefits to patients, healthcare …

Motivation In silico approaches often fail to utilize bioactivity data available for orthologous
targets due to insufficient evidence highlighting the benefit for such an approach. Deeper …

A program, MOLMAKER, is described which, in conjunction with a 2D−3D conversion program
and 3D database software, can generate de novo 3D databases to aid in drug design. …

Extracellular vesicles (EVs) have shown promise as biological delivery vehicles, but
therapeutic applications require efficient cargo loading. Here, we developed new methods for …

Precise genome editing using CRISPR-Cas9 is a promising therapeutic avenue for genetic
diseases, although off-target editing remains a significant safety concern. Guide RNAs …

Genome editing, specifically CRISPR/Cas9 technology, has revolutionized biomedical research
and offers potential cures for genetic diseases. Despite rapid progress, low efficiency of …

Prime editing recently emerged as a next-generation approach for precise genome editing.
Here we exploit DNA double-strand break (DSB) repair to develop two strategies that install …