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

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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Bibliographic Details
Main Authors: Suzan M. Hammond, Frank Abendroth, Larissa Goli, Jessica Stoodley, Matthew Burrell, George Thom, Ian Gurrell, Nina Ahlskog, Michael J. Gait, Matthew J.A. Wood, Carl I. Webster
Format: Article
Language:English
Published: American Society for Clinical investigation 2022-12-01
Series:JCI Insight
Subjects:
Neuroscience
Online Access:https://doi.org/10.1172/jci.insight.154142

Internet

https://doi.org/10.1172/jci.insight.154142

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