Archive for Nancy Fliesler
Cerebral adrenoleukodystrophy: How the Cooksons dodged a devastating disease
Heather Cookson believes that if she hadn’t insisted her son Ricky get a brain MRI to investigate his frequent headaches, neither he nor his younger brother Jerry would be walking or living full lives today. “I just wanted peace of mind,” she says. “Ricky got the MRI, and that’s when the lesion was found.” The ... Read More about Cerebral adrenoleukodystrophy: How the Cooksons dodged a devastating disease
Gene therapy for adrenoleukodystrophy: Studies find both risks and benefits
Cerebral adrenoleukodystrophy (CALD), portrayed in the film Lorenzo’s Oil, is a devastating disorder caused by a mutation on the X chromosome. Boys with CALD progressively lose their neurologic function and, if untreated, eventually become non-responsive. Most pass away within 10 years of diagnosis. Until recently, the only treatment for CALD was a blood (hematopoietic) stem ... Read More about Gene therapy for adrenoleukodystrophy: Studies find both risks and benefits
Shoring up heart muscle’s mini ‘managers’ to treat heart failure
Our heart muscle is studded with tiny dyads, intricately designed structures that manage incoming electrical signals and calcium release to coordinate our heartbeats. Could gene therapy help maintain dyads’ structure and boost the function of failing hearts? A new study suggests it can. “We know that in heart failure from many causes, dyads become disorganized,” says ... Read More about Shoring up heart muscle’s mini ‘managers’ to treat heart failure
EarlyBird: Addressing dyslexia through game play
Up to 10 percent of the population has dyslexia, yet many children are diagnosed only after struggling with reading for years. The stigma can cause low self-esteem, depression, and antisocial behavior. But imagine we could identify children at risk for dyslexia before they start formal reading instruction, then help them build the skills they need ... Read More about EarlyBird: Addressing dyslexia through game play
Will early intervention prevent asthma in school-age children?
Asthma affects about 1 in 10 children, often sending them to the emergency room or causing them to miss school. Allergic conditions in young children — like food allergies or eczema — are often a precursor. Susceptible children begin producing IgE antibodies, which trigger allergic reactions and spur the “allergic march” toward asthma. IgE antibodies ... Read More about Will early intervention prevent asthma in school-age children?
Curbing blood cancers by teaching immune cells to kill mutant stem cells
Blood stem cells, which give rise to all of our blood cell types, undergo a quality assurance process after they’re born. As the lab of Leonard Zon, MD, director of the Stem Cell Research program at Boston Children’s, has documented, immune cells known as macrophages interact with each newly born cell. They engulf and eat ... Read More about Curbing blood cancers by teaching immune cells to kill mutant stem cells
Will people accept a fentanyl vaccine? Interviews draw thoughtful responses
In 2022, more than 100,000 people died from opioid overdoses in the U.S., according to the National Center for Health Statistics. Roughly three quarters of these deaths involved fentanyl, a highly potent synthetic opioid. Fentanyl-related deaths have spiked over the past decade, many of them occurring in people with no known history of opioid use ... Read More about Will people accept a fentanyl vaccine? Interviews draw thoughtful responses
When community is as important as the science: Olúmídé Fagboyegun
In his short, prolific neuroscience career, Olúmídé Fagboyegun has always sought community. It’s served him well, from his years at community college in Maryland to his PhD work in the Stevens Lab at Boston Children’s Hospital. Landing in Maryland from his native Nigeria at age 15, Fagboyegun found he had to repeat part of high ... Read More about When community is as important as the science: Olúmídé Fagboyegun
A new anti-cancer strategy: Overriding tumor edits
Cancers are clever and often find ways to dodge people’s immune systems, making them hard to eradicate. Immunotherapies such as CAR-T cells and checkpoint inhibitors can sharpen the immune system’s attack and cure the cancer. But they don’t work for most solid tumors. We now know that tumors can edit their genes to evade immune ... Read More about A new anti-cancer strategy: Overriding tumor edits
Humble cells in a little-known organ manage brain inflammation
Deep in the brain, sheets of tissue known as the choroid plexus produce cerebrospinal fluid (CSF) and act as a protective barrier between the brain and CSF. But the lab of Maria Lehtinen, PhD, at Boston Children’s Hospital has shown that the little-known choroid plexus does much more. For example, it secretes factors that promote ... Read More about Humble cells in a little-known organ manage brain inflammation