Rare disease

Founding Member Harvey Lodish has been twice honored by the international science community for his path-breaking scientific accomplishments and intellectual leadership.

Using a mouse model, the lab of Whitehead Institute Founding Member Harvey Lodish, has now determined that combining the cholesterol-lowering drug fenofibrate with glucocorticoids could allow for dramatically lower steroid doses in the treatment of  Diamond Blackfan anemia and other erythropoietin-resistant anemias.

By directly altering the gene coding for the prion protein (PrP), Whitehead Institute researchers have created mouse models of two neurodegenerative prion diseases, each of which manifests in different regions of the brain.  These new models for fatal familial insomnia (FFI) and Creutzfeldt-Jakob disease (CJD) accurately reflect the distinct patterns of destruction caused by the these diseases in humans.  Remarkably, as different as each disease is, they both spontaneously generate infectious prions.

Researchers at Whitehead Institute have identified a key target protein of glucocorticoids, the drugs that are used to increase red blood cell production in patients with certain types of anemia, including those resulting from trauma, sepsis, malaria, kidney dialysis, and chemotherapy.