A vaccine to prevent opioid overdose?
Sharon Levy, MD, MPH, who directs the Adolescent Substance Use and Addiction Program (ASAP) at Boston Children’s Hospital, was getting her youngest son ready for school, when her husband Ofer, an infectious disease physician at the same hospital, came to her with an offer. The NIH was soliciting proposals to develop an opioid vaccine. Would ... Read More about A vaccine to prevent opioid overdose?
Presidential awardee explores epigenetics via slime molds, worms, and more
Pretty much all of your cells have the same DNA code. Yet they come in an astonishing number of forms. Some cells contract so that your heart can beat or your muscles flex. Others have jobs in your immune system, tuned to recognize different threats. And cells can change their behavior, altering their metabolism for ... Read More about Presidential awardee explores epigenetics via slime molds, worms, and more
Single-cell sequencing reveals glioblastoma’s shape-shifting nature
Glioblastoma, a cancer that arises in the brain’s supporting glial cells, is one of the worst diagnoses a child can receive. The grade IV, highly malignant tumor aggressively infiltrates healthy brain tissue, and most children die of the disease within one to two years of diagnosis, similar to adults. “The current approach is surgical removal, ... Read More about Single-cell sequencing reveals glioblastoma’s shape-shifting nature
Pupil dilation and heart rate, analyzed by AI, may offer early autism diagnosis
Autism and other neurodevelopmental disorders often aren’t diagnosed until a child is a few years of age, when behavioral interventions and speech/occupational therapy become less effective. But new research this week in PNAS suggests that two simple, quantifiable measures — spontaneous fluctuations in pupil dilation or heart rate — could enable much earlier diagnosis of ... Read More about Pupil dilation and heart rate, analyzed by AI, may offer early autism diagnosis
Tissue models and a gene therapy for a deadly heart arrhythmia
Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a leading cause of sudden death from cardiac arrest in children and young adults. Marked by racing, irregular heartbeats, the inherited arrhythmia is typically silent until a child suddenly collapses and faints, at an average age of 12. The trigger is an adrenaline surge, caused by exercise or emotional ... Read More about Tissue models and a gene therapy for a deadly heart arrhythmia
New stem cell pipeline makes more lifelike cell products
Researchers in many fields of science and medicine engineer different types of human cells for drug testing and other purposes. They often begin with stem cells — unspecialized cells that have the potential to form many different types of tissue. By turning on certain genes, the scientists attempt to coax the stem cells into becoming ... Read More about New stem cell pipeline makes more lifelike cell products
Optimized CRISPR/Cas9 gene editing averts hearing loss in ‘Beethoven’ mice
Using a novel gene-editing approach, scientists at Boston Children’s Hospital and Harvard Medical School have salvaged hearing in a mouse model of hereditary deafness, with no apparent off-target effects. The system successfully identified a single misspelled “letter” in the defective copy of a gene required for hearing, disabled this aberrant copy, and spared the healthy ... Read More about Optimized CRISPR/Cas9 gene editing averts hearing loss in ‘Beethoven’ mice
Overriding resistance to epigenetic inhibitors in neuroblastoma
Neuroblastoma and other children’s cancers pose unique challenges. They’re not caused by the same kinds of genetic mutations that cause adult cancers, and only a minority of their mutations can be targeted with drugs. In a recent study, a team led by Kimberly Stegmaier, MD, at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center systematically deleted every gene ... Read More about Overriding resistance to epigenetic inhibitors in neuroblastoma
First sharp images reveal structure of key inflammatory protein
After decades of attempts by the scientific community, researchers have now provided the first clear look at a protein implicated in a vast array of inflammatory conditions. The finding, published recently in Nature, lifts a blindfold that has hampered scientists’ ability to intervene when the immune system overreacts to perceived threats. The protein, known as ... Read More about First sharp images reveal structure of key inflammatory protein
‘Good’ bacteria may prevent – and reverse – food allergy
Food allergy is a large and growing public health problem. For reasons that remain a mystery, the number of Americans who suffer from the disease has risen sharply over the last decade to as many as 32 million, according to one recent estimate. Nearly 8 percent of children in the U.S. — about two in ... Read More about ‘Good’ bacteria may prevent – and reverse – food allergy