ANTISENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES
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United States of America
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Jan 23, 2020
app pub date -
Sep 5, 2019
filing date -
Sep 5, 2019
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Abstract
Alternative splicing events in SCN1A gene can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in SCN1A gene can modulate the expression level of functional proteins in Dravet Syndrome patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition caused by SCN1A, SCN8A or SCN5A protein deficiency.
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