Research
Taking a targeted approach when leukemia comes back
The news that your child has cancer always comes as a shock, but for one cancer, acute lymphoblastic leukemia (ALL), parents can take comfort in the fact that doctors are really good at treating it. The cure rate for ALL has, over the last 40 years, climbed to nearly 90 percent. Less comforting is the fact ... Read More about Taking a targeted approach when leukemia comes back
Research
Immune cells “sculpt” brain circuits — by eating excess connections
The image above and the movie below show an immune cell caught in the act of tending the brain. The cell below has just eaten away unnecessary connections, or synapses, between neurons. That’s not something these cells, known as microglia, were previously thought to do. As immune cells, it was thought that their job was ... Read More about Immune cells “sculpt” brain circuits — by eating excess connections
Clinical Care
Is rapamycin the new aspirin?
I’ve heard it said that if aspirin had to go through today’s FDA approval process, it would never be approved for over-the-counter use because it just does so many things. Lately, it’s been hard to cover biomedical research at Children’s without stumbling on another drug that’s also FDA-approved and also seems to have multiple uses: ... Read More about Is rapamycin the new aspirin?
Research
Gene-therapy trial will attempt to restore hearing in deaf mice
Sound waves produce the sensation of hearing by vibrating hair-like structures on the inner ear’s sensory hair cells. But how this mechanical motion gets converted into electrical signals that go to our brains has long been a mystery. Scientists have believed some undiscovered protein is involved. Such proteins have been identified for taste, smell and ... Read More about Gene-therapy trial will attempt to restore hearing in deaf mice
Research
Avoiding the needle: Engineering blood vessels to secrete drugs
People who rely on protein-based drugs often have to endure IV hookups or frequent injections, sometimes several times a week. And protein drugs – like Factor VIII and Factor IX for patients with hemophilia, alpha interferon for hepatitis C, interferon beta for multiple sclerosis — are very expensive. What if they could be made by people’s own ... Read More about Avoiding the needle: Engineering blood vessels to secrete drugs
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
Research
A new start for gene therapy for ‘bubble boy’ disease: First U.S.-treated patient doing well
Until this month, Agustín Cáceres’s baptism was the only time his family could come close to him. Everyone had to wear masks, gloves and gowns. After that, he went into isolation, along with his mother Marcela, who came out only for meals. His father Alberto, and his four-year-old brother Jeremías, kept to a separate bedroom. ... Read More about A new start for gene therapy for ‘bubble boy’ disease: First U.S.-treated patient doing well
Clinical Care
Moving gene therapy into high gear
Gene therapy, still experimental but beginning to enter the clinic, attempts to utilize advanced molecular methods to treat and even reverse genetic diseases. The field started in earnest about 25 years ago and has had many setbacks along the way to its recent earliest successes. International collaboration has been critical. Children’s Hospital Boston is one ... Read More about Moving gene therapy into high gear
Research
Brain juice and stem cells: Revisiting an ancient view of cerebrospinal fluid
Christopher Walsh, MD, PhD, is chief of Genetics and a Howard Hughes Medical Institute Investigator at Children’s Hospital Boston, where his research focuses on genes that regulate the development and function of the human cerebral cortex. Mutations in these genes are known to cause autism and epilepsy, as well as intellectual disabilities and other learning ... Read More about Brain juice and stem cells: Revisiting an ancient view of cerebrospinal fluid
Research
With algae blooms hope for a long-acting local anesthetic
For decades, Chile’s shoreline has had problems with periodic algal blooms – referred to as Red Tide, but actually containing a mix of microorganisms including bluegreen algae. Their toxins accumulate in shellfish, landing seafood consumers in the hospital, partially paralyzed, sometimes needing ventilators to breathe. The nerve block caused by the toxins is reversible, so ... Read More about With algae blooms hope for a long-acting local anesthetic