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BioWorld - Monday, March 23, 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 in neurons from Rett patients that produce some functional protein, correcting the altered gene expression and improving neuronal functions, and in vivo in mice.
BioWorld BioWorld Science Science Drug design, drug delivery and technologies Genetic/congenital Neurology/psychiatric Women's health

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