A 'David and Goliath' Protein Story (IMAGE)
Caption
Many proteins come in various versions, called isoforms. Chen Shen of the Vakoc lab was studying the long form of a protein called NSD3 - a 'Goliath' -- on the hunch that it might be involved in the regulation of genes that go awry in AML, an often fatal blood-cell cancer. She was surprised to discover that a seemingly uninteresting, much-truncated version of the protein called NSD3-short - 'David' -- was in fact playing a key role in the illness. It was a protein that the cancer depended upon; even more important, it proved to be the target of the promising new drug called JQ1, which Vakoc and colleagues have shown to be highly effective in treating animal models of AML. The drug is soon expected to enter Phase 2 trials in human patients.
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Vakoc Lab, CSHL
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