CRISPR-based system highlights new drug targets in a deadly leukemia

CRISPR-based system highlights new drug targets in a deadly leukemia


So transcription factors, if one looks at
the structure of their shape one finds that they lack deep, greasy crevices on
their surface. This is important because to design a drug to hit a protein
requires having a pocket with which to fit the drug into. And so transcription
factors unfortunately lack those classical druggable properties needed to
develop medicines in a simple fashion. My lab recently has identified a new
strategy for controlling a transcription factor that promotes acute myeloid
leukemia this transcription factor is called MEF2C and despite this
well-appreciated role in leukemia to date no one has identified a way to
target this protein with drugs. Using a technology involving the use of CRISPR
genome editing, we have discovered a approach to control
this MEF2C protein. This leads to a potential new therapeutic opportunity
for drug discovery, which we are intensely pursuing. But more broadly,
these efforts are leading us to try to apply this to many other types of cancer
in which transcription factors lie at the root cause.

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