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AssigneeThe Johns Hopkins University
InventorsIngie Hong, Yoichi Araki, Richard C. Johnson, Richard Huganir, Yinuo Han
Estimating the term…
This document relates to splice reporter nucleic acid constructs (e.g., splice reporter plasmids). For example, splice reporter nucleic acid constructs that can be used to (e.g., are designed to) screen for agents (e.g., anti-sense oligonucleotides (ASOs)) that can modulate SYNGAP1 splicing are provided. Also provided herein are methods and materials for modulating SYNGAP1 splicing. For example, agents that can modulate SYNGAP1 splicing (e.g., splice-switching ASOs (SSOs)) can be administered to a mammal (e.g., a human) having, or at risk of developing, a SYNGAP1-associated neurodevelopmental disorders (NDD) to increase expression of a SynGAP1-α1 polypeptide and/or a SynGAP1-α2 polypeptide in cells within that mammal (e.g., to treat the mammal).
A nucleic acid construct for identifying a SYNGAP1 splice event, said nucleic acid construct comprising:
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A method for modulating SYNGAP1 gene splicing in a cell, said method comprising:
A method for increasing a level of a SynGAP1-α1 polypeptide or a SynGAP1-α2 polypeptide in a cell, said method comprising, administering to said cell an SSO that targets a splice site within a SYNGAP1 gene.
A method for treating a mammal having or at risk of developing a SYNGAP1-associated neurodevelopmental disorder (NDD), wherein said method comprises:
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Dependencies are inferred from the claim text.