New drug pathway linked with tuberous sclerosis
Tuberous sclerosis complex (TSC) is a neurological disorder causing non-cancerous tumors, called cortical tubers, to grow throughout the brain and body, as well as other conditions like epilepsy and autism. While medications are used to treat some of the manifestations of the disease, safe and more effective treatments targeting disease at a fundamental level are ... Read More
Type III interferon in COVID-19: Protective or harmful?
Our immune system makes interferons and other cytokines to help us fight viruses. But in COVID-19, we’ve learned that they can also contribute to damaging, potentially life-threatening lung inflammation. New work published yesterday in the journal Science helps tease out the good from the bad for one interferon of interest: type III. Key takeaways. At ... Read More
Creating hairy human skin: Not as easy as you think
Key takeaways· Scientists have created the first cultured human skin capable of growing hair embedded with fat and nerve cells. · Potential applications include burn treatments, and testing medications and cosmetics· The skin organoid system developed is a proof of concept for creating other human organoids, such as the inner ear. For more than 40 ... Read More
Gene therapy with a new base editing technique restores hearing in mice
Using a new genetic engineering technique, known as base editing, researchers from Boston Children’s Hospital and the Broad Institute of MIT and Harvard, have restored hearing in mice with a known recessive genetic mutation. Key takeaways· This is the first example of repairing a recessive gene mutation.· Repairing a single mutation in the Tmc1 gene restored partial hearing in ... Read More
Tracking an organism’s development, cell by cell
A new mouse model allows scientists to track every cell in the body — from the embryo stage until adulthood. The system is the first of its kind and should yield a greater understanding of development, aging, and disease. Scientists described it last week in the journal Cell. “The dream of many developmental biologists for ... Read More
Antibodies in the gut: Roles in autoimmune disease, food allergy… and COVID-19?
From our tonsils through our intestines, the lining of our gastrointestinal tract forms a border between our body and the outer world. More than a static tissue, it is an active tissue constantly bombarded by exposure to foods, viruses, and bacteria. It houses more than 40 trillion of its own bacteria, known as the microbiome. ... Read More
Disulfiram inhibits inflammatory gatekeeper protein: Could it be helpful in COVID-19?
Inflammation is the alarm system by which cells first respond to potential danger. But in excess, inflammation can be deadly. In new research from the Program in Cellular and Molecular Medicine (PCCM) at Boston Children’s Hospital, scientists discovered that disulfiram, an FDA-approved drug commonly used for treating alcoholism, blocks a key gatekeeper protein involved in ... Read More
A drug treatment for telomere diseases?
For years, Donna Martin carried a piece of scrap paper with the words “dyskeratosis congenita,” which she believed might explain her son Brad’s sudden, mysterious affliction. A routine blood test had revealed Brad’s bone marrow was failing, unable to keep up with his need for healthy blood cells. His condition, Donna knew, would worsen over ... Read More
Gasdermin E: A new approach to cancer immunotherapy
Tumors have figured out various ways to prevent the immune system from attacking them. Medicine, for its part, has fought back with cancer immunotherapy. The major approach uses checkpoint inhibitors, drugs that help the immune system recognize cancer cells as foreign. Another method, CAR T-cell therapy, directly engineers peoples’ T cells to efficiently recognize cancer ... Read More
Designing a coronavirus vaccine for next year – and the years beyond
As the number of coronavirus infections swell daily across the globe, strategies for developing a safe and effective vaccine are rapidly moving forward. In response to this public health crisis, the Precision Vaccines Program (PVP) at Boston Children’s Hospital is on the front lines of developing a coronavirus vaccine targeted especially toward older populations, those ... Read More