Article

Transcriptional changes in non-human primate tissues after intrathecal delivery of serotype 9 adeno-associated viral vector: insights into organ toxicities

2025-05-14

Abstract excerpt

<h4>ABSTRACT</h4> Adeno associated virus (AAV)-based gene transfer has brought transformative therapeutic benefits to patients with otherwise untreatable genetic diseases. However, treatment-related organ toxicities, particularly for high doses, remain a safety concern in the clinic with some translatability in preclinical species. In the present study, we conducted an RNAseq analysis in non-human primates admini...

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Literature Corpus work: b04e4568-062f-5399-9d93-0b0d8236a451
DOI: 10.1101/2025.05.09.652903

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Transcriptional changes in non-human primate tissues after intrathecal delivery of serotype 9 adeno-associated viral vector: insights into organ toxicitiesDOI 10.1101/2025.05.09.652903Read

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