Article

Systemic antibody-oligonucleotide delivery to the central nervous system ameliorates mouse models of spinal muscular atrophy

2021-07-29

Abstract excerpt

Antisense oligonucleotides (ASOs) have emerged as one of the most innovative new genetic drug modalities, however, the high molecular weight limits their bioavailability for otherwise treatable neurological disorders. We investigated conjugation of ASOs to an antibody against the murine transferrin receptor (TfR), 8D3 130 , and evaluated it via systemic administration in mouse models of the neurodegenerative dise...

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Literature Corpus work: 13cad521-f8e1-5425-a1ce-93c5a3645c14
DOI: 10.1101/2021.07.29.454272

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