Sickle cell gene therapy shows promise in search for cure [Video]

CLEVELAND — 34-year-old Danielle Lee says she remembers most of her young life in and out of Cleveland hospitals every time she had a painful sickle cell episode.  As the years went on those hospital stays became longer and the pain became progressively worse, despite the standard protocols of treatment. 

The disease took her spleen, gall bladder and damaged her hip joints and bones.  So when she was offered an opportunity to try a new gene editing therapy that is a one time treatment that re-engineers her stem cells to correct the mutation that caused her sickle cell disease, she signed up. 

She knew the treatment couldn’t fix the damage already done, but she never thought she’d hear the words functional cure. 

“It’s such a blessing, God is good, that’s all I can say,” Danielle said.