Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy

Antisense oligonucleotides (ASOs) have emerged as one of the most innovative new genetic drug modalities. However, their high molecular weight limits their bioavailability for otherwise-treatable neurological disorders. We investigated conjugation of ASOs to an antibody against the murine transferri...

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Bibliografiske detaljer
Udgivet i:JCI Insight
Main Authors: Hammond, Suzan M., Abendroth, Frank, Goli, Larissa, Stoodley, Jessica, Burrell, Matthew, Thom, George, Gurrell, Ian, Ahlskog, Nina, Gait, Michael J., Wood, Matthew J.A., Webster, Carl I.
Format: Artigo
Sprog:Inglês
Udgivet: American Society for Clinical Investigation 2022
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7614086/
https://ncbi.nlm.nih.gov/pubmed/36346674
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.154142
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