METHOD OF REGULATING GENE EXPRESSION

A method to regulate patient gene expression by controllably circulating antisense oligonucleotide material (ASO) through a closed fluid circuit formed between the patient's ventricle and lumbar regions. To that end, after coupling a fluid channel between those regions, such embodiments add ASO...

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Bibliographic Details
Main Authors PATEL, Rajan, RICCARDI, Gianna, SIOPES, William, X., Jr, DEPASQUA, Anthony, GLICKMAN, Marcie, KALISH, Kevin, VOSE, Joshua, G
Format Patent
LanguageEnglish
French
German
Published 01.05.2024
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Summary:A method to regulate patient gene expression by controllably circulating antisense oligonucleotide material (ASO) through a closed fluid circuit formed between the patient's ventricle and lumbar regions. To that end, after coupling a fluid channel between those regions, such embodiments add ASO to the fluid channel (preferably after the channel is primed with CSF) and energize a pump to controllably flow the CSF and the ASO mixed with the CSF. CSF/ASO fluid flow may be managed to localize treatment (e.g., providing deep brain distribution) while minimizing toxicity potentially caused by the ASO to certain nerves (e.g., the peripheral nerve).
Bibliography:Application Number: EP20220829302