Mutations during prenatal development may contribute to schizophrenia
Schizophrenia is known to have a genetic component, and variants in 10 genes have been identified as markedly increasing schizophrenia risk. But together, these genes account for under 5 percent of cases. Now, a pilot study in the journal Science suggests another important contributor to schizophrenia: distinctive patterns of non-inherited (somatic) mutations. These mutations appear ... Read More about Mutations during prenatal development may contribute to schizophrenia
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
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
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
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
Creating the next generation of mRNA vaccines
During the COVID-19 pandemic, mRNA vaccines came to the rescue, developed in record time and saving lives worldwide. Researchers in the Precision Vaccines Program at Boston Children’s Hospital have developed two novel technologies that could make these and future mRNA vaccines more potent and longer-lasting — at smaller doses and with fewer side effects. The ... Read More about Creating the next generation of mRNA vaccines
Tracking influenza in its first battleground: The nose
The answer to curbing influenza could be right under our noses — or, more accurately, inside them. New research maps happenings in the nose during the course of influenza in exquisite detail, and could potentially lead to new targets and more effective nasal flu vaccines. The nose is often the gateway to respiratory infections, where ... Read More about Tracking influenza in its first battleground: The nose
From ‘hit to vial’: Discovery and optimization of a promising vaccine adjuvant
Many vaccines are only partially effective, have waning efficacy, or do not work well in the very young or the very old. For more than a decade, Ofer Levy, MD, PhD, and David Dowling, PhD, in the Precision Vaccines Program at Boston Children’s Hospital, have tried improving vaccines by adding compounds known as adjuvants to ... Read More about From ‘hit to vial’: Discovery and optimization of a promising vaccine adjuvant