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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, 8D3\n                    <jats:sub>130<\/jats:sub>\n                    , and evaluated it via systemic administration in mouse models of the neurodegenerative disease spinal muscular atrophy (SMA). SMA, like several other neurological and neuromuscular diseases, is treatable with single-stranded ASOs that modulate splicing of the survival motor neuron 2 (\n                    <jats:italic>SMN2<\/jats:italic>\n                    ) gene. Administration of 8D3\n                    <jats:sub>130<\/jats:sub>\n                    -ASO conjugate resulted in elevated levels of bioavailability to the brain. 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Systemic delivery of nucleic acid therapies with brain-targeting antibodies offers powerful translational potential for future treatments of neuromuscular and neurodegenerative diseases.\n                  <\/jats:p>","DOI":"10.1172\/jci.insight.154142","type":"journal-article","created":{"date-parts":[[2022,11,8]],"date-time":"2022-11-08T12:00:46Z","timestamp":1667908846000},"source":"Crossref","is-referenced-by-count":73,"title":["Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy"],"prefix":"10.1172","volume":"7","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5640-7569","authenticated-orcid":true,"given":"Suzan M.","family":"Hammond","sequence":"first","affiliation":[{"name":"Department of Paediatrics, John Radcliffe Hospital, and"},{"name":"MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, United 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