We are so honored and excited to announce that we won the JumpStart Research Tools Matching Grant through The Orphan Disease Center (ODC) at the University of Pennsylvania! In partnership with the Coriell Institute, this grant will support the creation of high-quality, Shwachman-Diamond Syndrome patient-derived induced pluripotent stem cell lines (iPSCs). The breakthrough technology of iPSCs has quickly become an important tool for modeling and investigating human disease, screening drugs, and providing an unlimited supply of human tissue for research, and we jumped at the opportunity to bring this tool to the SDS research community.
Partnering with the ODC and the Coriell Institute will ensure that the iPSCs are the
highest quality
biobanking and storage are managed professionally
distribution of the cells is fast and efficient, globally
The first phase of the project is set to start this fall, and the iPSCs are expected to be available to researchers – anywhere in the world – as soon as mid-2023.
Your donations to our “Expand the Toolbox” fundraising campaign made this project possible, turning hope into action. With the JumpStart Grant, we were able to leverage your donations three fold!
In the second phase of this project, we will further amplify the impact of the iPSCs by creating isogenic pairs. When researchers are testing therapeutics on cells, they need to prove that the effect they see is directly related to SDS (i.e. SBDS mutations), and is not just a random effect. The best way to show this is to treat two cell lines in parallel - one that harbors SDS mutations, and one doesn’t (but is otherwise all the same). If the effect is SDS specific, it should be much stronger in the SDS cells. To create the isogenic pairs, we will contract leading SDS and iPSC experts. Details to follow. But we need your financial support to fund this work and to leverage additional funding sources. Let’s turn hope into action and “Expand the SDS Research Toolbox” together.
This project is part of our roadmap. With your support, we are making steady progress every day.
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