Archive for stem cells
Avoiding a lifetime of injections: Can gene editing cure severe congenital neutropenia?
Fionn Mulrooney, a cheerful 11-month-old, in Plymouth, Massachusetts, has no idea he has a life-threatening genetic disease. Nor does he seem fazed by the daily subcutaneous injections his parents have learned how to give him. And little does he know that cells from his bone marrow are helping scientists develop an innovative gene-editing approach that ... Read More about Avoiding a lifetime of injections: Can gene editing cure severe congenital neutropenia?
How do patients with cystic fibrosis respond to COVID-19? An ‘airway in a dish’ may give answers
So far, based on clinical data, patients with cystic fibrosis (CF) don’t appear to be especially susceptible to COVID-19. And when they do get infected, they don’t seem to get sicker. But Ruobing (Ruby) Wang, MD, who cares for patients with CF in the Division of Pulmonary Medicine at Boston Children’s Hospital, thinks there is ... Read More about How do patients with cystic fibrosis respond to COVID-19? An ‘airway in a dish’ may give answers
Therapy developed at Boston Children’s stops preeclampsia before it starts
Preeclampsia occurs in about 3 to 5 percent of all pregnancies. Characterized by very high maternal blood pressure, it can lead to serious, sometimes fatal, complications in both mother and baby. In severe cases, early delivery is often the only effective treatment, usually before the baby’s lungs are fully developed. Researchers within the Division of ... Read More about Therapy developed at Boston Children’s stops preeclampsia before it starts
Lung ‘organoids’ capture early-stage lung cancer; could help test treatments
Lung cancer, the leading cancer killer in the U.S., is often missed in its earlier stages. And while recent imaging advances offer earlier detection, early-stage lung cancers still have no targeted treatments. Key takeaways Mini “organoids” made from lung cells offer a rapid platform for tracking early-stage lung cancers and for testing possible treatments. RNA ... Read More about Lung ‘organoids’ capture early-stage lung cancer; could help test treatments
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 about Creating hairy human skin: Not as easy as you think
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 about Tracking an organism’s development, cell by cell
Long-term hemophilia treatment could lie in patients’ own cells
Children (and adults) with hemophilia are slow to form blood clots, so are at constant risk for uncontrolled bleeding. Even when the skin isn’t broken, a fall or a simple toe stub can become a serious medical issue: internal bleeding cause permanent damage to muscles and joints. While regularly replacing the missing or malfunctioning clotting ... Read More about Long-term hemophilia treatment could lie in patients’ own cells
Following blood stem cells in the body could yield better, safer treatments
Blood stem cells make all the different kinds of blood and immune cells in our body. Scientists have long been trying to make these cells in the lab and use them to make different blood cell types on demand. Mastering these feats could help make bone marrow transplants more efficient and less toxic, and could ... Read More about Following blood stem cells in the body could yield better, safer treatments
Going into science: Women scientists at Boston Children’s offer advice to girls
In honor of the International Day of Women and Girls in Science (February 11), we invited women scientists at all stages of their careers at Boston Children’s Hospital to share their scientific agendas. Here is some of what they had to say. The scientists also offered their advice for girls interested in entering the field. ... Read More about Going into science: Women scientists at Boston Children’s offer advice to girls
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