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

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

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 possib...

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Veröffentlicht in:Human gene therapy. Part B. Methods 2018-08, Vol.29 (4), p.189-199
Hauptverfasser: Chansel-Debordeaux, Lucie, Bourdenx, Mathieu, Dutheil, Nathalie, Dovero, Sandra, Canron, Marie-Helene, Jimenez, Clement, Bezard, Erwan, Dehay, Benjamin
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container_end_page 199
container_issue 4
container_start_page 189
container_title Human gene therapy. Part B. Methods
container_volume 29
creator Chansel-Debordeaux, Lucie
Bourdenx, Mathieu
Dutheil, Nathalie
Dovero, Sandra
Canron, Marie-Helene
Jimenez, Clement
Bezard, Erwan
Dehay, Benjamin
description 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.
doi_str_mv 10.1089/hgtb.2017.192.r3
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title Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2/9 and scAAV2/rh10 Variants in Newborn Rats
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