Nucleoside therapy for telomere diseases
Awardee: Agarwal Suneet
Institution: Boston Children's Hospital
Grant Amount: $63,000.00
Funding Period: February 1, 2024 - January 31, 2025
Summary:
Telomere biology disorders (TBDs) are a spectrum of diseases characterized by life-threatening complications including bone marrow failure, liver and lung disease. Tissue and organ transplantation remain the primary treatments, but cures that address the underlying problem and restore telomere length to improve symptoms throughout the body are lacking. We recently discovered that molecules called nucleosides can increase telomere length in human cells, including those from patients with TBDs. This is exciting because nucleosides have already been used in children with another rare disease, and appear to be safe and effective for that disease. In this proposal, we will now rigorously test whether these same nucleosides can effectively increase telomere length in human stem cells, both in the dish and in animal models. If successful, these experiments will provide critical results that will help take nucleosides into clinical trials for patients with TBDs.