Delving into the causes of attention deficits: Childhood adversity, lost sleep, and dopamine
New research on the effects of adversity in childhood ties together stress, sleep loss, and attention deficits later in life. It also uncovers some of the underlying brain biology and potential treatment approaches — while revealing a puzzling sex-specific effect. The lab of Takao Hensch, PhD, has long studied time windows during development — commonly ... Read More about Delving into the causes of attention deficits: Childhood adversity, lost sleep, and dopamine
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?
Novel procedure may bridge the treatment gap for symptomatic flexible flatfoot
Most children develop arches in their feet by early adolescence. About 20 percent, however, have pes planovalgus (PPV), also known as flexible flatfoot. Most children with PPV can participate in sports and other activities without issue, but others have ongoing pain in their feet that limit function and activities. Until recently in the U.S., children ... Read More about Novel procedure may bridge the treatment gap for symptomatic flexible flatfoot
Finding a possible genetic treatment for rare arrhythmias
Variants in a gene that plays a key role in heart function can cause potentially life-threatening arrhythmia syndromes known as calmodulinopathy. Calmodulinopathy is rare and causes arrhythmias that are poorly treated by current options. Boston Children’s cardiologist William Pu, MD, believes he has found a promising custom genetic treatment: antisense oligonucleotides that deplete the disease-causing gene product. ... Read More about Finding a possible genetic treatment for rare arrhythmias
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
In cases of pediatric cervical spine instability, two attending surgeons are better than one
Cervical spine instability (caused by congenital deformity, trauma, or a tumor) can affect children in a number of ways: from chronic headaches to bowel dysfunction to severe mobility limitations. In many cases, misaligned vertebrae compress the spinal cord and surrounding nerves, causing neurological issues, which, if left untreated, can lead to long-term disability. Common indications ... Read More about In cases of pediatric cervical spine instability, two attending surgeons are better than one
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