Mapping cells to create targeted treatments for interstitial lung disease
John Kennedy, MD, MSc, remembers the relative simplicity of his first genetic mapping project. In a Harvard Medical School lab, he helped map a gene for the neurological disease mucolipidosis type IV in less than a year. “I was fresh out of college. I thought with the global momentum of the Human Genome Project, we were going to ... Read More about Mapping cells to create targeted treatments for interstitial lung disease
Blood across our lifetimes: An age-specific ‘atlas’ tells a dynamic story
The stem cells that form our blood, also known as hematopoietic stem cells (HSCs), are with us throughout our lives. A new study reveals how HSCs ramp up and pivot their activities depending on the body’s needs at the time, from before we’re born until old age. Researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders ... Read More about Blood across our lifetimes: An age-specific ‘atlas’ tells a dynamic story
AI-enabled medical devices are burgeoning, but many haven’t been tested in children
Medical devices that incorporate artificial intelligence and machine learning are proliferating. In 2013, the FDA approved fewer than 10 such devices; by 2023, this number had grown to nearly 250. Devices include software to help interpret radiology images, wearable devices that monitor the heart and flag risks, and devices that analyze brain signals for potential ... Read More about AI-enabled medical devices are burgeoning, but many haven’t been tested in children
Years of experience support the safe use of fine-needle aspiration for pediatric thyroid nodules
Ultrasound-guided fine-needle aspiration (FNA) has long been used as a safe and effective way to evaluate thyroid nodules in adults, usually without sedation. Less is known about the safety and tolerability of FNA in children, although small studies suggest that the approach can be performed without sedation and with minimal complications. Now, a report by ... Read More about Years of experience support the safe use of fine-needle aspiration for pediatric thyroid nodules
Changing lives through genetics: The Children’s Rare Disease Collaborative
A 14-year-old girl was having back pain after a car accident and visited an orthopedic clinic at Boston Children’s Hospital. In the course of her care, she joined the Children’s Rare Disease Collaborative (CRDC), a hospital-wide effort to enroll children and adults with rare diseases in genetic studies. Genetic testing revealed that both she and ... Read More about Changing lives through genetics: The Children’s Rare Disease Collaborative
Model enables study of age-specific responses to COVID mRNA vaccines in a dish
mRNA vaccines clearly saved lives during the COVID-19 pandemic, but several studies suggest that older people had a somewhat reduced immune response to the vaccines when compared with younger adults. Why? Researchers at Boston Children’s Hospital, led by Byron Brook, PhD, David Dowling, PhD, and Ofer Levy, MD, PhD, found some answers — while providing ... Read More about Model enables study of age-specific responses to COVID mRNA vaccines in a dish
Could a pill treat sickle cell disease?
The new gene therapies for sickle cell disease — including the gene-editing treatment Casgevy, based on research at Boston Children’s Hospital — have been game-changing for the patients who have received them. But Stuart Orkin, MD, the Boston Children’s hematologist whose work led the way to Casgevy, wants to go even further. “The editing therapy ... Read More about Could a pill treat sickle cell disease?
A sickle cell first: Base editing, a new form of gene therapy, leaves Branden feeling ‘more than fine’
Though he doesn’t remember it, Branden Baptiste had his first sickle cell crisis at age 2. Through elementary school, he was in and out of the hospital with pain episodes, not knowing why. As he got older, he learned he had sickle cell disease. His red blood cells were forming sickle shapes and getting stuck ... Read More about A sickle cell first: Base editing, a new form of gene therapy, leaves Branden feeling ‘more than fine’
Injected microbubbles could be a safe way to deliver emergency oxygen
For years, researchers and clinicians have been trying to find a way to rapidly deliver oxygen to patients when traditional means of oxygenation are difficult or ineffective during critical moments of cardiac or respiratory arrest. Sometimes, hypoxemia caused by airway obstruction or lung disease can be so severe that methods to boost low-oxygen levels (including ... Read More about Injected microbubbles could be a safe way to deliver emergency oxygen
A universal gene therapy for Diamond-Blackfan anemia is poised for clinical testing
Diamond-Blackfan anemia (DBA), first described at Boston Children’s Hospital in 1938, is a rare blood disorder in which the bone marrow cannot make mature, functioning red blood cells. Children with this life-threatening anemia have few treatment options. A small handful with a well-matched donor can be cured with bone marrow transplant, but most rely on ... Read More about A universal gene therapy for Diamond-Blackfan anemia is poised for clinical testing