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The California Institute for Regenerative Medicine has awarded MATTHEW PORTEUS, associate professor of pediatrics, a grant of $5.2 million to lay the groundwork for a clinical trial of a possible treatment for sickle cell disease.
Porteus has shown that he can take human blood stem cells with the gene defect that causes sickle cell disease and use gene-editing tools to repair the faulty gene. He also showed that he could successfully transplant those repaired blood stem cells into mice.
“We are extremely excited that, with CIRM support, we may be able to use gene correction to treat this terrible disease,” Porteus said.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.