Stories about: stimulus funding

Sickle cell: New looks at a neglected disease

sickle cell disease red blood cells
(OpenStax College/Wikimedia Commons)

Sickle-cell anemia was the first disease to have its genetic cause identified, in the 1950s — a milestone in human genetics. Yet today, there’s just one FDA-approved drug, hydroxyurea, developed 20 years ago at Children’s. Though it’s a mainstay of treatment, reducing the frequency of severe pain, acute chest syndrome and the need for blood transfusions, it can cause toxicity, and about half of patients aren’t helped by it. Only a hematopoietic stem-cell transplant is curative.

Research on sickle-cell disease has generally been underfunded compared with other genetic diseases like cystic fibrosis that aren’t as common. But Children’s has been exploring new treatment approaches for decades, and two exciting possibilities have emerged.

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Helping disease cells handle the stress of reprogramming

Fanconi anemia cells have multiple defects in response to DNA damage, making it hard to create iPS cells from them.

I’m attending an international pediatric oncology meeting in Boston with about 2,000 other people. In a session on gene therapy, David Williams, chief of Hematology/ Oncology at Children’s, talked about trying to treat a rare inherited bone marrow failure syndrome, Fanconi anemia, by correcting the gene in the patient’s blood stem cells. It didn’t work the first time, but he has a new tactic.

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Thinking outside the box in diabetes

Diabetes prevalence and mean particulate (PM2.5) concentration, Southeast U.S., 2005.

Two heads are better than one in solving a problem. And sometimes it’s better to skip over the usual suspects. Crowdsourcing, or contracting out problems to large groups, can often provide fresh leads on intractable problems – especially when those problems are pressing.

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Recovery Act funding boosts science, jobs—but what’s next?

Last week marked the beginning of the end of the biggest boost in federal research funding in U.S. history. September 30 was the deadline for the National Institutes of Health and other federal agencies to commit the last stimulus dollars for science allotted by the American Recovery and Reinvestment Act of 2009. The looming drop in funding now has researchers and organizations wondering how to keep people in their labs employed and their projects going.

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