A very interesting study, but obviously in the pre-clinical, in vitro phase. In addition to showing that you can turn off genes on Chr. 21 (similar to X inactivation) in actual human beings, I suspect that the demonstrating when such a treatment could have benefit is incredibly important. Many of the physical conditions (e.g., heart defects, GI issues) and developmental delays (e.g., intellectual, motor, coordination) that we see in individuals with Down syndrome have their foundations very early in utero development. So, while a treatment based on Chr. 21 inactivation might have some mild effects when given to a newborn or young child with Down syndrome (such as giving them a “boost” to do even better in Early Intervention programs), I suspect that real gains would require intervention during the first trimester, which seems like a huge challenge.
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