Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2/9 and scAAV2/rh10 Variants in Newborn Rats.
Human Gene Therapy Methods. 2018-08-01; 29(4): 189-199
DOI: 10.1089/hgtb.2017.192.r3
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1. Hum Gene Ther Methods. 2018 Aug;29(4):189-199. doi: 10.1089/hgtb.2017.192.r3.
Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2/9 and
scAAV2/rh10 Variants in Newborn Rats.
Chansel-Debordeaux L(1)(2)(3), Bourdenx M(1)(2), Dutheil N(1)(2), Dovero S(1)(2),
Canron MH(1)(2), Jimenez C(1)(2)(3), Bezard E(1)(2), Dehay B(1)(2).
Author information:
(1)1 Univ. Bordeaux, Institut des Maladies Neurodégénératives , UMR 5293, F-33000
Bordeaux, France .
(2)2 CNRS, Institut des Maladies Neurodégénératives , UMR 5293, F-33000 Bordeaux,
France .
(3)3 CHU Bordeaux , Service de Biologie de la reproduction-CECOS, F-33000
Bordeaux, France .
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: 10.1089/hgtb.2017.192.r3
PMID: 30064266