Porteus receives $1M stem cell grant for ‘bubble boy disease’

The grant will allow the researcher to pursue a stem-cell-based gene therapy approach to correcting a form of severe combined immunodeficiency in humans.

Matthew Porteus

Matthew Porteus, PhD, associate professor of pediatrics at the School of Medicine, was awarded $1 million today from the California Institute for Regenerative Medicine to pursue a stem-cell-based gene therapy approach to correcting a form of severe combined immunodeficiency (also known as SCID, or “bubble boy disease”) in humans.

The award was part of the state stem cell agency’s Preclinical Development Awards. Seven awards were granted, for a total of $25.2 million. The agency said in a statement that the awards support “projects that use or target stem cells, have shown potential in early stage research and are now ready to move one step closer to clinical trials in people.”

“This investment will let us further test the early promise shown by these projects,” said Jonathan Thomas, PhD, JD, head of the agency’s governing board, in the statement. “Preclinical work is vital in examining the feasibility, potential effectiveness and safety of a therapy before we try it on people. These projects all showed compelling evidence that they could be tremendously beneficial to patients. We want to help them build on that earlier research and move the projects to the next level.”

With this award, Stanford has received a total of around $297 million from CIRM.


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