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Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2/9 and scAAV2/rh10 Variants in Newborn Rats

Abstract : Abstract 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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https://www.hal.inserm.fr/inserm-02439491
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Submitted on : Sunday, March 28, 2021 - 10:42:59 AM
Last modification on : Monday, March 29, 2021 - 8:51:58 AM
Long-term archiving on: : Tuesday, June 29, 2021 - 6:05:00 PM

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Lucie Chansel-Debordeaux, Mathieu Bourdenx, Nathalie Dutheil, Sandra Dovero, Marie-Helene Canron, et al.. Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2/9 and scAAV2/rh10 Variants in Newborn Rats. Human gene therapy methods, New Rochelle, NY : Mary Ann Liebert, 2018, 29 (4), pp.189-199. ⟨10.1089/hgtb.2017.192.r3⟩. ⟨inserm-02439491⟩

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