After A Life Of Painful Sickle Cell Disease, A Patient Hopes Gene-Editing Can Help

Thursday, October 10, 2019

From NPR, heard on Morning Edition

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Victoria Gray slides open a closet door, pulls out a suitcase and starts packing piles of clothes.

"My goodness," says Gray. "Did I really bring all this?"

Gray, who has sickle cell disease, is the first patient with a genetic disorder who doctors in the United States have tried to treat using the powerful gene-editing technique CRISPR.

Today, Gray is getting ready to finally go home to Forest, Miss., after months away from her four children so she could undergo the experimental treatment, which involves infusions of genetically-modified bone marrow cells.

"I'm very excited," says Gray, who has been living in a temporary apartment in Nashville, Tenn., for several weeks since getting discharged from the nearby TriStar Centennial Medical Center. "I know it's going to be emotional for me. I miss the hugs and the kisses and just everything."

NPR has exclusive access to chronicle Gray's journey through the highly anticipated clinical trial.

"Victoria is a pioneer in this. And we are very excited," says her doctor, Haydar Frangoul.

"I feel hopeful for the future," Gray says.