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BioWorld - Thursday, March 5, 2026
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Genetic/congenital

Alternative splicing strategy shows promise for Rett syndrome

March 4, 2026
By Mar de Miguel
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A therapeutic strategy based on alternative splicing of the MECP2 gene could restore protein levels in Rett syndrome, a neurological disorder caused by mutations in that gene. Scientists at Baylor College of Medicine have successfully tested this approach both in vitro and in vivo in a mouse model that produces some functional protein, correcting the altered gene expression and improving neuronal functions.
BioWorld BioWorld Science Science Drug design, drug delivery and technologies Genetic/congenital Neurology/psychiatric Women's health

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