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US 2026/0218192 A1Published application
Compositions and methods for treatment of monogenic neurodevelopment disorders
AssigneePYC Therapeutics Limited
InventorsAnja STIRNWEISS, Dulce Vargas LANDIN, Jessica NICHOLS, Janya GRAINOK, Adam MARTIN
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Abstract
Described herein are antisense oligonucleotides (ASOs), vectors, and related compositions and methods for increasing endogenous expression of SHANK3 protein and uses thereof for conditions associated with SHANK3 haploinsufficiency such as Phelan-McDermid syndrome.
Claims9
1-64. (canceled)
An antisense oligonucleotide (ASO), wherein the nucleotide sequence of the antisense oligonucleotide consists of SEQ ID NO:1092, and wherein each nucleotide of the ASO comprises a 2′-O-methoxyethyl moiety.
Dependencies are inferred from the claim text.