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Antisense oligonucleotides as a feasible therapy to treat MECP2 duplication syndrome
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Antisense oligonucleotides as a feasible therapy to treat MECP2 duplication syndrome

by Texas Children’s Hospital  Credit: CC0 Public Domain Many cognitive neurodevelopmental disorders are a result of too many or too few copies of certain genes or chromosomes. To date, no treatment options exist for this class of disorders. MECP2 duplication syndrome (MDS) is one such disorder that primarily affects boys and results from a duplication...