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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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Detalhes bibliográficos
Publicado no: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.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2022
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Acesso em linha: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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