ATXN2 IRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING ATXN2-ASSOCIATED NEURODEGENERATIVE DISEASES
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United States of America Patent
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Issued Date -
Sep 5, 2024
app pub date -
Jul 28, 2021
filing date -
Jul 29, 2020
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Abstract
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an ATXN2 gene, as well as methods of inhibiting expression of an ATXN2 gene and methods of treating subjects having an ATXN2-associated neurodegenerative disease or disorder, e.g., SCAs and ALS, using such dsRNAi agents and compositions.
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