ORLANDO, Fla. -

Little Hunter Miller's motor is always running. Like most toddlers, he's sometimes one step away from trouble, but for Hunter, being rough and tumble can have serious side effects. Hunter has severe hemophilia.

Three days after he was born, a routine circumcision caused a major scare.

"You know, a baby gets up in the morning and their diapers are just full," said Hunter's grandmother, Tina Miller. "Well, his was full, but it was full of blood."

Doctors diagnosed Hunter with hemophilia A, which means his blood is missing a protein, known as clotting factor VIII. When he gets hurt, doctors need to inject the clotting factor to stop the bleeding. He's had eight emergency room visits in 19 months.

"Him falling, bumping his head too hard, little cuts. He cut the roof of his mouth with a tortilla chip and that was a hospital trip," said Heather Frederick, Hunter's mother.

Dr. Katherine Ponder studies gene therapy treatment for hemophilia and other blood disorders. Her lab treated hemophilia A in animals, but she said the therapy isn't quite ready for humans yet.

"I think that the big question is going to be the safety," said Katherine Ponder, hematologist at Washington University School of Medicine in St. Louis.

But gene therapy has proven effective for some patients with hemophilia B. Researchers at St. Jude's Children's Research Hospital and University College of London have added the missing protein -- factor IX -- to a specially-engineered virus, which travels to the patients liver and transfers the gene.

"This modifies the disease from a situation where they might bleed once a week to a situation where they hardly ever bleed," Ponder explained.

See the original post:
Health Beat: Gene therapy: From bench to bedside: Hemophilia

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