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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| Autors principals: | , , , , , , , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
American Society for Clinical investigation
2022-12-01
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| Col·lecció: | JCI Insight |
| Matèries: | |
| Accés en línia: | https://doi.org/10.1172/jci.insight.154142 |
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