“(The researchers) hope this one-two punch used in combination will overcome the genetic mutations that cause DMD, restore a missing protein needed for proper muscle function and allow those affected by the disease to lead relatively normal lives.”
“Their youngest son, Dylan, 11, was diagnosed with DMD in 2004. While he’s still ambulatory – many DMD patients require the use of wheelchairs by about age 10 – Dylan can no longer run or climb stairs and he can’t shoot a basketball over his head like other boys his age. Despite these challenges, Miceli said Dylan remains a happy, funny and engaged boy, full of life and passion.
“'We entered into this field because of the diagnosis of our son, but we hope our research can help many others,' she said. 'There are drugs that can help manage the symptoms of the disease, but nothing that changes its course dramatically. We’re trying to correct the defect that causes DMD with highly personalized genetic medicine.'”
"Clearly there is still a long way to go before we know if this approach will work in people but we're delighted that funding from the stem cell agency is helping the researchers move their work forward....This is what voters set out to do when they approved Proposition 71 to create the stem cell agency."