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

8 Nov 2022

Abstract excerpt

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 transferrin receptor, 8D3130, and evaluated it via systemic administration in mouse models of the neurodegenerative disease...

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PMID: 36346674
DOI: 10.1172/jci.insight.154142

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