Archive for stem cells
Motor neurons made from patients’ cells reveal possible ALS drugs and targets
Amyotrophic lateral sclerosis (ALS) is a severe, fatal neurodegenerative disorder causing loss of motor neurons and voluntary muscle action. While mouse studies have identified potential treatments, these drugs have typically done very poorly in human trials. “One of the most difficult challenges in drug discovery is identifying a target that has a key role in ... Read More
Recommendations for reproducibility in stem cell research
The ability to program induced pluripotent stem cells (iPSCs) and drive their differentiation into a variety of neural cells is essential for studying neurological disorders, including intellectual and developmental disabilities (IDDs). But issues regarding variability and reproducibility from lab to lab make translating discoveries difficult, in turn slowing the progress of IDD research. To address ... Read More
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
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
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
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
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
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
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
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