Article
Quantifying and mitigating motor phenotypes induced by antisense oligonucleotides in the central nervous system.
Molecular therapy : the journal of the American Society of Gene Therapy - 4 Dec 2024
Moazami Michael P, Rembetsy-Brown Julia M, Sarli Samantha L, McEachern Holly R, Wang Feng, Ohara Masahiro, Wagh Atish, Kelly Karen, Krishnamurthy Pranathi Meda, Weiss Alexandra, Marosfoi Miklos, King Robert M, Motwani Mona, Gray-Edwards Heather, Fitzgerald Katherine A, Brown Robert H, Watts Jonathan K
Abstract excerpt
Antisense oligonucleotides (ASOs) are emerging as a promising class of therapeutics for neurological diseases. When injected directly into cerebrospinal fluid, ASOs distribute broadly across brain regions and exert long-lasting therapeutic effects. However, many phosphorothioate (PS)-modified gapmer ASOs show transient motor phenotypes when injected into the cerebrospinal fluid, ranging from reduced motor...
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