Potential drug treatments for telomere diseases

April 24, 2020

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 stem cells will increase tissue regenerative capacity in the blood, lungs, and other organs affected in DC and other diseases.”

The researchers’ findings were published in the journal Cell Stem Cell.

Read the full story on the Boston Children’s Hospital website.

Source article 

Nagpal, N. et al. (2020). Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells. Cell Stem Cell. DOI: 10.1016/j.stem.2020.03.016