Key result
Single-dose intracardiac administration of rAAV2/9 and rAAV2/rh10 vectors in newborn rats achieved brain and peripheral transduction with predominant neuronal tropism and varying expression patterns.
Single-dose intracardiac administration of rAAV2/9 and rAAV2/rh10 vectors in newborn rats results in widespread transduction with predominant neuronal tropism.
Intracardiac AAV delivery may enable neonatal CNS transduction; leaves open clinical translation pending safety and efficacy data in larger models.
Recombinant adeno-associated virus serotype 9 (rAAV2/9) and pseudotype rhesus-10 (rAAV2/rh10) are used for gene delivery, especially into the central nervous system. Both serotypes cross the blood-brain barrier and mediate stable long-term transduction in dividing and nondividing cells. Among possible routes of administration, intracardiac injection holds the potential for widespread vector diffusion associated with a relatively simple approach. In this study adopting the intracardiac route, we compare the cell-specific tropism and transfection efficacy of a panel of engineered rAAV2/9 and rAAV2/rh10 vectors encoding the enhanced green fluorescent protein. We observed transduction in the brain and peripherally, with a predominant neuronal tropism while the various serotypes achieved different expression patterns.
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Chansel‐Debordeaux et al. (2018) studied this question. scAAV2/9 and scAAV2/rh10 variants was evaluated on Cell-specific tropism and transfection efficacy. Single-dose intracardiac administration of rAAV2/9 and rAAV2/rh10 vectors in newborn rats achieved brain and peripheral transduction with predominant neuronal tropism and varying expression patterns.
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