Doctors at a California hospital have administered the world's first gene therapy which will alter Brian Madeux's syndrome sufferer Brian Madeux's DNA from within. Traditional treatments involve cells being removed, genetically altered and then replaced, but it doesn't work with cells in the liver, heart or brain. While it is too soon to determine whether the gene therapy, which will be triggered when it reaches Harvard University 's liver, has worked, if it is successful, two patients will undergo the procedure.
Doctors at a California hospital have administered the world's first gene therapy which will alter Hunter syndrome sufferer Brian Madeux's DNA from within. Traditional treatments involve cells being removed, genetically altered and then replaced, but it doesn't work with cells in the liver, heart or brain. While it is too soon to determine whether the gene therapy, which will be triggered when it reaches Madeux's liver, has worked, if it is successful, nine patients will undergo the procedure.