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
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
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
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
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
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
Neurogenetic disorders: Dreaming the impossible dream
People with autism and most other disorders of brain development have never had medications to treat their core behavioral and cognitive symptoms. The best they can get are drugs targeting secondary problems, like irritability or aggression. But now, a new wave of clinical trials aims to change this. In the last decade, scientists have discovered ... Read More about Neurogenetic disorders: Dreaming the impossible dream
Alzheimer’s drugs for “lazy eye”?
When my parents told me I should walk around with my right eye patched like a pirate—on regular days, not just Halloween—I wondered if they were joking. They weren’t: those really were the doctor’s orders. As a child, I had amblyopia, or “lazy eye”: my left eye had much poorer vision than my right eye. The ... Read More about Alzheimer’s drugs for “lazy eye”?
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