Gene therapy for hearing loss: Tag-teaming from the lab to the clinic
Two-year-old Miles is one of the first patients with hereditary hearing loss to receive gene therapy at Boston Children’s Hospital — to striking effect. In May 2025, Miles received an injection delivering a healthy copy of a gene called OTOF into the cochlea of his inner ear. Now, Miles is thriving. He’s had no adverse ... Read More about Gene therapy for hearing loss: Tag-teaming from the lab to the clinic
The journey to a treatment for hereditary spastic paraplegia
In 2016, Darius Ebrahimi-Fakhari, MD, PhD, then a neurology fellow at Boston Children’s Hospital, met two little girls with spasticity and decreased muscle tone in their legs, which affected their walking. Both girls, Robbie Edwards and Molly Duffy, had been diagnosed with hereditary spastic paraplegia (HSP), which comprises a group of more than 80 genetic ... Read More about The journey to a treatment for hereditary spastic paraplegia
A toast to BRD4: How acidity changes the immune response
It started with wine. Or more precisely, a conversation about it. “My colleagues and I were talking about how some people think drinking wine may be anti-inflammatory,” recalls Xu Zhou, PhD, from the Division of Gastroenterology, Hepatology, and Nutrition at Boston Children’s Hospital. “There’s no scientific ground for that, but we know wine is acidic.” ... Read More about A toast to BRD4: How acidity changes the immune response
A safe, pain-specific anesthetic shows preclinical promise
All current local anesthetics block sensory signals — pain — but they also interrupt motor signals, which can be problematic. For example, too much epidural anesthesia can prevent mothers in labor from being able to push. Prolonged local anesthesia after orthopedic surgery can leave patients unable to participate in rehab. Researchers at Boston Children’s Hospital ... Read More about A safe, pain-specific anesthetic shows preclinical promise
A unique marker for pericytes could help forge a new path for pulmonary hypertension care
Pulmonary arterial hypertension (PAH) is a rare condition that’s difficult to treat. The hallmarks of the disease — narrowing of the arterioles and capillaries that deliver blood to the lungs — force the heart to work harder. In severe cases, PAH can lead to heart failure. Pericytes support capillary function and may play a role ... Read More about A unique marker for pericytes could help forge a new path for pulmonary hypertension care
New genetic insights could change how we treat, and talk about, polycystic ovary syndrome
Polycystic ovary syndrome (PCOS) has long been viewed as a hormonal disorder affecting women of reproductive age. However, ongoing research led by Jia Zhu, MD, attending physician in the Division of Endocrinology, and her colleagues is changing that. Their most recent findings indicate that PCOS is part of a broader metabolic and reproductive disorder that ... Read More about New genetic insights could change how we treat, and talk about, polycystic ovary syndrome