MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING POMPE DISEASE
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Issued Date -
Sep 5, 2024
app pub date -
Jun 17, 2022
filing date -
Jun 21, 2021
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Abstract
Aspects of the disclosure relate to oligonucleotides (e.g., RNAi oligonucleotides such as siRNAs) designed to target GYSI RNAs and targeting complexes for delivering the oligonucleotides to cells (e.g., muscle cells) and uses thereof, particularly uses relating to treatment of disease (e.g., Pompe Disease).
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