New sicklecelldisease genetherapies depend on getting the right mouse
article, Mitchell Weiss and colleagues from St. Jude Children's Research Hospital, Memphis, U.S., have investigated a promising new treatment that is being developed in Weiss's lab, and works by editing genes to switch on the production of this healthy, fetal hemoglobin in adult.
Surprisingly, 70% of the Berkeley mice died from the therapy, and it only activated production of the healing fetal hemoglobin gene in 3.1% of mouse stem cells. In contrast, the experimental treatment activated the fetal hemoglobin gene in 57% of red blood cells in the Townes mice and did not affect the animals' survival.
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