COMPOSITIONS FOR MODULATING MECP2 EXPRESSION

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United States of America Patent

SERIAL NO

15554409

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Disclosed herein are compounds and methods for decreasing methyl CpG binding protein 2 (MECP2) mRNA and protein expression. Such compounds and methods are useful to treat, prevent, or ameliorate MECP2 associated disorders and syndromes. Such MECP2 associated disorders include MECP2 duplication syndrome. In certain embodiments, compounds useful for modulating expression and amount of MECP2 mRNA and protein are antisense compounds. In certain embodiments, the antisense compounds are modified antisense oligonucleotides. In certain embodiments, the antisense compounds are single-stranded antisense oligonucleotides. In certain embodiments, the antisense compounds are not siRNA compounds.

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Patent Owner(s)

Patent OwnerAddress
IONIS PHARMACEUTICALS INCCARLSBAD CA 92010

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Freier, Susan M San Diego, US 526 6555

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