Archive for stem cells
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 about Forty years waiting for a cure: ALD gene therapy trial shows early promise
Tagged: adrenoleukodystrophy, gene therapy, stem cells
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 about New Human Neuron Core to analyze ‘disease in a dish’
Tagged: autism research, epilepsy, neuroscience, psychiatry, stem cells
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 about Stem cell medicine gets a “roadmap” and a quality assurance tool
Tagged: genetics and genomics, regeneration, stem cells
‘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 about ‘Heart on a chip’ suggests a surprising treatment for a rare genetic disease
Tagged: cardiomyopathy, heart, organoids, orphan diseases, rare disease, stem cells
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 about Brain juice and stem cells: Revisiting an ancient view of cerebrospinal fluid
Tagged: neuroscience, proteomics, stem cells
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 about Messenger RNA: A game-changing advance in regenerative medicine