Archive for genetics and genomics
Orphan diseases: Bringing academia, industry, and government into the game
“If you build it, he will come,” the ghosts of baseball players past tell a farmer in Field of Dreams. But it’s not that easy. To put people in the seats you have to have all of the right pieces: the right team, including players and managers; the right park, one that works for both ... Read More about Orphan diseases: Bringing academia, industry, and government into the game
The neurology resident that could
Elizabeth Engle used to wait in peoples’ driveways until midnight, hoping to enroll them in her genetic studies of eye-movement disorders. She landed there by chance: during her neurology residency, she saw a little boy whose eyes were frozen in a downward gaze. Wanting to find a solution to a disorder that others had written ... Read More about The neurology resident that could
Childhood brain cancer: Learning to divide and conquer
Diversity is good in populations of people, but when it comes to cancer, it’s bad news. In the case of medulloblastoma—the most common malignant brain cancer in children—tumor diversity has been one of the greatest barriers to designing effective treatments. Now, in the largest genomic study of human medulloblastomas ever, Children’s researchers and their collaborators ... Read More about Childhood brain cancer: Learning to divide and conquer
Messenger RNA: A game-changing advance in regenerative medicine
A new way to reprogram ordinary human cells into stem cells, using RNAs, appears safer and much more efficient than current methods — and can much more readily transform stem cells into specialized cells to treat disease. We now know that scientists can take one of your cells, perhaps a skin cell, and transform it into ... Read More about Messenger RNA: A game-changing advance in regenerative medicine