Recovery from sickle cell disease brings a mixture of anxiety and hope

But she has other concerns, especially about her 7- and 12-year-old daughters. The long hospitalization for gene therapy followed by months of recovery would be difficult for them, she said.

“They’re not babies, but they need me,” Ms Polanco said.

Patients ask when in the course of the disease gene therapy is needed. If they wait too long, the disease can lead to a stroke or permanent damage to organs and bones. But if the disease is mild and manageable, is it better to wait? Andre Marcel Harris, 33, who lives in Houston, decided waiting was his best option.

“I’m not as sick as many others,” he said. “At this point, gene therapy is not part of the conversation.”

Shamar Lewis, 20, who lives in Orlando, Florida, has several worries — the possible long-term side effects of gene therapy, the costs, and the months-long treatment and recovery process.

Yet sickle cell disease has been such a burden. He spent years in his early teens unable to attend school because he was so sick – his school even sent teachers to his home. He has now finished high school and wants to join the army, but has since learned that with sickle cell disease he would not be accepted. With gene therapy, “I could really live my life,” he told his mother, Carla Lewis.

But what would that life be like?

Without the disease, said Teonna Woolford, 31, a sickle cell patient and activist who lives in Owings Mills, Maryland, patients “don’t know who they are.” Many worry, she said, about “the swapping of a familiar problem – sickle cell disease – with something else and the general fear of the unknown”.