DOSING OF siRNA COMPOUNDS TO THE CISTERNA MAGNA
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United States of America Patent
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Issued Date -
May 30, 2024
app pub date -
Mar 4, 2022
filing date -
Mar 5, 2021
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Abstract
One aspect of the present invention relates to a double stranded iRNA agent comprising an antisense strand which is complementary to a target gene and one or more lipophilic moieties conjugated to one or more internal positions on at least one strand, which provides for targeting to, and uptake by, tissues and cells of the CNS via administration to a subarachnoid space, e.g., the cisterna magna. Another aspect of the invention relates to a method of gene silencing in tissues and cells of the CNS, that includes administering to the subarachnoid space of a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs.
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