Antisense oligonucleotides as a feasible therapy to treat MECP2 duplication syndrome
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4 March 2021Antisense oligonucleotides as a feasible therapy to treat MECP2 duplication syndrome
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 spanning the methyl-CpG binding protein 2 (MECP2) locus located on the X chromosome.
