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<i>In vivo</i> selection in non-human primates identifies superior AAV capsids for on-target CSF delivery to spinal cord

2023-09-13

Abstract excerpt

Systemic administration of adeno-associated virus (AAV) vectors for spinal cord gene therapy has challenges including toxicity at high doses and pre-existing immunity that reduces efficacy. Intrathecal delivery of AAV vectors into the cerebral spinal fluid (CSF) can avoid many of the issues of systemic delivery, although achieving broad distribution of the vector and transgene expression throughout the spinal cord...

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Identifiers and source

Literature Corpus work
3e040c3c-1ac7-5546-874f-9fb73e536a99
DOI
10.1101/2023.09.13.557506
Open publication

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<i>In vivo</i> selection in non-human primates identifies superior AAV capsids for on-target CSF delivery to spinal cordDOI 10.1101/2023.09.13.557506
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