Tag Archives: Insertion

Question?: Rett Syndrome

John asks…

Why are diseases that are found to be curable/reversible in mice?

not in humans?

Serious answers only please.

Asking b/c Rett Syndrome ( a devastating neurological disorder) was completely reversed in mice like 3 years ao & I don’t understand why the same basic concepts that made that possible can’t be used in living humans suffering from this horrible disease

OCTOBER IS RETT SYNDROME AWARENESS MONTH
Care Today-Cure tommorow !

admin answers:

Great question and thanks for posting about Rett Syndrome and helping raise awareness!

In the experiments on mice (1 1/2 yr ago) they silenced the MECP2 in the mice (with insertion of a “stop cassette”), and then un-silenced it to see if the effects would be permanent like they believed they would be. So the mice didn’t actually have Rett Syndrome and in RS there is no “stop cassette” built into it to turn on again. So the real outcome of this study showed that once a cure/treatment is found, these girls could possibly be normal again and not neurologically damaged permanently…this is amazing in itself but doesn’t mean we are any closer to a cure.

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