Archive for stem cells
Genetics drive deep investigations into blood cell production
Knowing how different kinds of blood cells form from their stem cell progenitors can shed light on blood disorders and aid in finding new treatments. A series of recent studies co-led by researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center applied a variety of genetic tools to provide new insights on blood cell production. ... Read More
Blood stem cell transplants from any donor, without toxicity?
Many blood disorders, immune disorders and metabolic disorders can be cured with a transplant of hematopoietic (blood-forming) stem cells, also known as bone marrow transplant. But patients must first receive high-dose, whole-body chemotherapy and/or radiation to deplete their own defective stem cells, providing space for the donor cells to engraft. These “conditioning” regimens are highly ... Read More
Medical milestone: Making blood stem cells in the lab
Pluripotent stem cells can make virtually every cell type in the body. But until now, one type has remained elusive: blood stem cells, the source of our entire complement of blood cells. Since human embryonic stem cells (ES cells) were isolated in 1998, scientists have tried to get them to make blood stem cells. In ... Read More
Stem cell workaround cracks open new leads in Diamond Blackfan anemia
Diamond Blackfan anemia (DBA) has long been a disease waiting for a cure. First described in 1938 by Louis K. Diamond, MD, of Boston Children’s Hospital and his mentor, Kenneth Blackfan, MD, the rare, severe blood disorder prevents the bone marrow from making enough red blood cells. It’s been linked to mutations affecting a variety of ... Read More
Forty years waiting for a cure: ALD gene therapy trial shows early promise
A small piece of notepaper, folded twice, sits tucked in a slot of the secretary desk in the living room. Every so often, I pull it out, read it, then reread. Addressed to my mom, the paper has a question and two boxes, one “yes” and one “no,” written with the careful precision of a ... Read More
New Human Neuron Core to analyze ‘disease in a dish’
Last week was a good week for neuroscience. Boston Children’s Hospital received nearly $2.2 million from the Massachusetts Life Sciences Center (MLSC) to create a Human Neuron Core. The facility will allow researchers at Boston Children’s and beyond to study neurodevelopmental, psychiatric and neurological disorders directly in living, functioning neurons made from patients with these ... Read More
Stem cell medicine gets a “roadmap” and a quality assurance tool
If you’ve lost your way on the Boston subway, you need only consult a map to find the best route to your destination. Now stem cell engineers have a similar map to guide the making of cells and tissues for disease modeling, drug testing and regenerative medicine. It’s a computer algorithm known as CellNet. As ... Read More
‘Heart on a chip’ suggests a surprising treatment for a rare genetic disease
It was the variability that intrigued pediatric cardiologist William Pu, MD, about his patient with heart failure. The boy suffered from a rare genetic mitochondrial disorder called Barth syndrome. While he ultimately needed a heart transplant, his heart function seemed to vary day-to-day, consistent with reports in the medical literature. “Often patients present in infancy with severe ... Read More
Brain juice and stem cells: Revisiting an ancient view of cerebrospinal fluid
Christopher Walsh, MD, PhD, is chief of Genetics and a Howard Hughes Medical Institute Investigator at Children’s Hospital Boston, where his research focuses on genes that regulate the development and function of the human cerebral cortex. Mutations in these genes are known to cause autism and epilepsy, as well as intellectual disabilities and other learning ... Read More
Messenger RNA: A game-changing advance in regenerative medicine
A new way to reprogram ordinary human cells into stem cells, using RNAs, appears safer and much more efficient than current methods — and can much more readily transform stem cells into specialized cells to treat disease. We now know that scientists can take one of your cells, perhaps a skin cell, and transform it into ... Read More