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 E-Mail 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. A preclinical study published from the laboratory of Dr. Huda Zoghbi, professor at Baylor College of Medicine and director of the Jan and Dan Duncan Neurological Research Institute at Texas Children s Hospital, provides experimental evidence that supports the use of antisense oligonucleotides as a feasible strategy to treat MDS. The study also offers crucial insights into the pharmacodynamics of this approach, which will serve as an important guide for the design and implementation of future clinical trials for this disorder. The study appears in the journal

United statesBaylor college of medicineAlexanderj trostleSameers bajikarQi wangFrank rigoHuda zoghbiYehezkel sztainbergPaymaan jafar nejadZhandong liuJianrong tangYingyao shaoRegeneron pharmaceuticalsChao family foundationHamill foundationNational institutes of health