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Using red blood cells modified to carry disease-specific antigens, a team of scientists from Whitehead Institute and Boston Children’s Hospital have prevented and alleviated two autoimmune diseases—multiple sclerosis (MS) and type 1 diabetes—in early stage mouse models.  This research is an exciting step toward therapeutics for autoimmune diseases, which affect an estimated 23 million Americans.

Investigators at Whitehead Institute and the Broad Institute have succeeded in identifying the set of essential genes—those required for cellular proliferation and survival—in each of 14 human acute myeloid leukemia (AML) cell lines that had previously been characterized by genome sequencing. By combining their “gene essentiality map” with the existing genomic information, their study revealed liabilities in genetically defined subset of cancers that could be exploited for new therapies.

Parkinson’s disease (PD) and other “synucleinopathies” are known to be linked to the misfolding of alpha-synuclein protein in neurons. Less clear is how this misfolding relates to the growing number of genes implicated in PD through analysis of human genetics. Two new studies from researchers affiliated with Whitehead Institute and Massachusetts Institute of Technology explain how they used a suite of novel biological and computational methods to shed light on the question.

The use of proteasome inhibitors to treat cancer has been greatly limited by the ability of cancer cells to develop resistance to these drugs. But Whitehead Institute researchers have found a mechanism underlying this resistance--a mechanism that naturally occurs in many diverse cancer types and that may expose vulnerabilities to drugs that spur the natural cell-death process.

Whitehead researchers provide insight into a specific gene pathway that appears to regulate the growth, structure, and organization of the human cortex. They also demonstrate that 3D human cerebral organoids--miniature, lab-grown versions of specific brain structures--can be effective in modeling the molecular, cellular, and anatomical processes of human brain development. And they suggest a new path for identifying the cells affected by Zika virus.

Whitehead Institute researchers have determined how the master transcriptional regulator of the heat shock response, known as heat shock factor 1 (HSF1), is controlled in yeast. Understanding how HSF1 works, how it is regulated, and how to fine tune it in a cell-type specific way could lead to therapies for cancer and neurodegenerative diseases.

Susan Lee Lindquist, Ph.D., Member and former Director of Whitehead Institute, and one of the nation’s most lauded scientists, yesterday succumbed to cancer. Her nearly 40-year career was defined by intellectually courageous, boundary-defying research and a passion for nurturing new generations of scientific talent. 

“Sue has meant so much to Whitehead as an institution of science, and as a community of scientists, and her passing leaves us diminished in so many ways,” reflects David C. Page, M.D., Director of Whitehead Institute and Professor of Biology at the Massachusetts Institute of Technology (MIT). “She was a risk-taker and an innovator. She believed that if we were not reaching for things beyond our grasp, we were not doing our job as researchers; if we were not constantly striving for that which we could only imagine, we were not fulfilling our obligations to society as scientists.”