Making ionocytes: A step toward cell or gene therapy for cystic fibrosis
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators were a breakthrough for cystic fibrosis, improving the movement of chloride and water and moistening mucus secretions. But these drugs are expensive, don’t work in all patients with cystic fibrosis, and have side effects and interactions with other drugs. People who do respond to CFTR modulators must take ... Read More about Making ionocytes: A step toward cell or gene therapy for cystic fibrosis
Study shows young adults don’t easily transition to self-care of diabetes
Diabetes is challenging to manage at any age, but even more so for young adults who are handling the condition on their own for the first time. They need to transition from pediatric to adult care as smoothly as possible — but a recent study finds this isn’t happening. Research led by Katharine Garvey, MD, MPH, ... Read More about Study shows young adults don’t easily transition to self-care of diabetes
Girls and women in sports win the medal of lifelong health
Title IX, the law that prohibits sports discrimination on the basis of sex, has transformed sports for girls and women. Before its passage in 1972, only 295,000 female athletes participated in U.S. high school sports. Today that number is 3.2 million. In that same period, the number of female college athletes swelled from 30,000 to ... Read More about Girls and women in sports win the medal of lifelong health
Going out of the box to tackle pancreatic cancer
Pancreatic cancers are deadly and hard to treat, in part because they are so often detected at an advanced stage; overall five-year survival rates are about 11 percent. Two separate labs at Boston Children’s Hospital took out-of-the-box approaches to this difficult cancer, and both uncovered some very promising leads. Wiping out pancreatic tumors’ immune defense ... Read More about Going out of the box to tackle pancreatic cancer
Could gene therapy relieve post-hemorrhagic hydrocephalus?
Premature infants, especially very low birthweight babies, are at risk for intraventricular hemorrhage. A frequent complication of these brain bleeds is hydrocephalus, an accumulation of cerebrospinal fluid (CSF) in the brain ventricles that can gravely disrupt brain development. If hydrocephalus develops, a child may need shunt operations throughout life to manage the fluid buildup. Could ... Read More about Could gene therapy relieve post-hemorrhagic hydrocephalus?
A promising target for ALS and other neurodegenerative disorders: Curbing inflammation
When cells recognize a danger, such as an infection, they activate the innate immune system. Sentinel molecules sound an alarm, recruiting immune cells to take down the threat. In 2016, Judy Lieberman, MD, PhD and her colleagues at Boston Children’s Hospital showed that a protein called gasdermin D initiates a final, decisive step: pyroptosis, a ... Read More about A promising target for ALS and other neurodegenerative disorders: Curbing inflammation
Why do so many dementia treatments fail? Questioning mouse models of tau accumulation
To date, the search for effective treatments for dementia has yielded only disappointments. Many recent drug candidates target the tau protein, which aggregates and forms tangles in patients’ brain tissue and is involved in 75 percent of all dementias. While tau-targeting drugs have looked promising in mouse models, they’ve failed in clinical trials. A recent ... Read More about Why do so many dementia treatments fail? Questioning mouse models of tau accumulation
Study seeks to identify household triggers for chronic lung disease in children
Home is where the heart is, but it’s also where air pollutants, allergens, and other irritants can make breathing difficult for children with the chronic lung disease bronchopulmonary dysplasia (BPD). BPD is the most common respiratory disease affecting children who were born prematurely. It leads to impaired lung function and respiratory symptoms similar to those associated ... Read More about Study seeks to identify household triggers for chronic lung disease in children
Treating brain ‘hotspots’ and networks to address autism, aggression, and more
What if doctors could break down conditions like autism into their key symptoms, map these symptoms to “hotspots” in the brain, and then treat those areas directly with brain stimulation? If it bears out, such an approach could turn the care of neurologic and developmental disorders on its head, focusing on symptoms that are shared ... Read More about Treating brain ‘hotspots’ and networks to address autism, aggression, and more
Hip pain is different in female dancers: New insights from dynamic ultrasound
Dancers put unique demands on their hips, achieving extreme ranges of motion that can strain the joints and damage supporting tissues around them. Not surprisingly, hip injuries account for up to 17 percent of injuries in dancers and 27 percent among professional dancers. Microinstability — no small problem for dancers Conditions such as hip dysplasia ... Read More about Hip pain is different in female dancers: New insights from dynamic ultrasound