NCI Center for Cancer Research – Restoring Critical Genes
NCI Center for Cancer Research posted on LinkedIn:
“RESTORING CRITICAL GENES: Duy Anh “Harry” Le was just 2 months old when his parents first noticed strange bleeding spots developing on his body. In 2016, he was diagnosed with Wiskott-Aldrich Syndrome, a rare disease that affects white blood cell and platelet function. Harry’s father undertook a worldwide search for treatments at that point, eventually bringing his son to the U.S. in November 2016 to participate in a gene therapy clinical trial run by Sung-Yun Pai.
After the trial, Harry’s symptoms improved dramatically, and he is now a happy and healthy 9-year-old with a broad smile. “The clinical trial, it’s like a miracle. Now Harry can have a normal life, the same as other children,” says Duy.
“We followed each patient for at least five years, and they did great. Unhindered by their condition, they were able to act like kids again.” Dr. Pai says.
Read more about this research milestone.”
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