HSCI researchers have identified potential drugs to treat telomere diseases such as dyskeratosis congenita (DC), in which cells age prematurely. The researchers tested over 100,000 compounds on patient stem cells, and identified several drugs that restored the cells’ telomeres — the protective caps on the ends of chromosomes that regulate how cells age.
“We envision these to be a new class of oral medicines that target stem cells throughout the body,” said Suneet Agarwal, an HSCI principal faculty member at Boston Children’s Hospital. “We expect restoring telomeres in...
Read more about Potential drug treatments for telomere diseases
Using a difficult laboratory technique, HSCI physician-researchers have isolated genetically healthy stem cells from patients with Pearson Marrow Pancreas Syndrome (PS), a generally fatal infant blood disorder with less than a hundred reported cases worldwide. Children with PS experience a range of symptoms, most...