CRISPR base editing screens enable analysis of disease-associated variants at scale;
however, variable efficiency and precision confounds the assessment of variant-induced …

The advent of targeted therapies has led to tremendous improvements in treatment options
and their outcomes in the field of oncology. Yet, many cancers outsmart precision drugs by …

Proteostasis can be disturbed by mutations affecting folding and stability of the encoded
protein. An example is the ubiquitin ligase Parkin, where gene variants result in autosomal …

Continual advancements in genomics have led to an ever-widening disparity between the
rate of discovery of genetic variants and our current understanding of their functions and …

DNA methyltransferase 3A (DNMT3A) is a de novo cytosine methyltransferase responsible
for establishing proper DNA methylation during mammalian development. Loss-of-function …

Personalized cancer therapeutics bring directed treatment options to patients based on their
tumor's genetic signature. Unfortunately, tumor genomes are remarkably adaptable, and …

Altered metabolism is a hallmark of cancer; however, it has been difficult to specifically target
metabolism in cancer for therapeutic benefit. Cancers with genetically defined defects in …

A major challenge in human genomics is to decipher the context specific relationship of
sequence to function. However, existing tools for locus specific hypermutation and evolution …

UM171 is a potent small molecule agonist of ex vivo human hematopoietic stem cell (HSC)
self-renewal, a process that is tightly controlled by epigenetic regulation. By co-opting …

Mammalian hematopoiesis, the process by which blood cells develop from a common
progenitor cell, is evolutionarily conserved and unfolds continuously over the lifetime of an …