New Kind of ‘Designer’ Immune Cells Clear Baby’s Leukemia

A baby whom doctors thought almost certain to die has been cleared of a previously incurable leukemia in the first human use of an “off-the-shelf” cell therapy from Cellectis that creates designer immune cells.

One-year-old Layla had run out of all other treatment options when doctors at Britain’s Great Ormond Street Hospital (GOSH) gave her the highly experimental, genetically edited cells in a tiny 1-milliliter intravenous infusion.

Two months later, she was cancer-free and she is now home from hospital, the doctors said at a briefing about her case in London on Wednesday . . .

The gene-edited cell treatment was prepared by scientists at GOSH and University College London (UCL) together with the French biotech firm Cellectis, which is now funding full clinical trials of the therapy due to start next year . . .

Using a gene-editing technology called TALEN, which acts as “molecular scissors,” specific genes are then cut to make the T-cells behave in two specific ways: Firstly, they are rendered invisible to a powerful leukemia drug that would usually kill them and secondly they are reprogrammed to only target and fight against leukemia cells. (Read more from “New Kind of ‘Designer’ Immune Cells Clear Baby’s Leukemia” HERE)

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