Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
AssigneesCold Spring Harbor Laboratory, Stoke Therapeutics, Inc.
InventorsIsabel Aznarez, Huw M. Nash, Adrian Krainer
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Abstract
Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.
Claims30
1-188. (canceled)
A pharmaceutical composition comprising an antisense oligomer (ASO) or a vector encoding the ASO, wherein the ASO is complementary to a targeted portion of a retained-intron-containing pre-mRNA (RIC pre-mRNA) that encodes a target protein, wherein the RIC pre-mRNA comprises a retained intron, an exon flanking a 5′ splice site of the retained intron, and an exon flanking a 3′ splice site of the retained intron, wherein the ASO induces constitutive splicing of the retained intron from the RIC pre-mRNA encoding the target protein, and wherein the target protein is Ras/Rap GTPase-activating protein SynGAP (SYNGAP1) or Nav1.1.
Dependencies are inferred from the claim text.