Abstract
Liguore and colleagues characterize biodistribution of AAV-PHP.B in the rodent and rhesus macaque CNS following intra-CSF and intravascular delivery modalities. Although equal transduction efficiency was seen in mice regardless of delivery route, intra-cisterna magna infusion led to superior transduction efficiency compared with intra-carotid artery infusion in the rhesus macaque CNS.
Original language | English (US) |
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Pages (from-to) | 2018-2037 |
Number of pages | 20 |
Journal | Molecular Therapy |
Volume | 27 |
Issue number | 11 |
DOIs | |
State | Published - Nov 6 2019 |
Keywords
- AAV-PHP.B
- AAV9
- CNS
- adeno-associated virus
- brain
- gene therapy
- gene transfer
- mouse
- rhesus macaque
- viral transduction
ASJC Scopus subject areas
- Molecular Medicine
- Molecular Biology
- Genetics
- Pharmacology
- Drug Discovery