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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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:JCI Insight
Egile Nagusiak: 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.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society for Clinical Investigation 2022
Gaiak:
Research Article
Sarrera elektronikoa: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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