1. Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2/9 and scAAV2/rh10 Variants in Newborn Rats
- Author
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Nathalie Dutheil, Erwan Bezard, Sandra Dovero, Mathieu Bourdenx, Benjamin Dehay, Lucie Chansel-Debordeaux, Clement Jimenez, Marie-Hélène Canron, Institut des Maladies Neurodégénératives [Bordeaux] (IMN), Université de Bordeaux (UB)-Centre National de la Recherche Scientifique (CNRS), Service de Biologie de la Reproduction - CECOS |Bordeaux], CHU Bordeaux [Bordeaux], and Dehay, Benjamin
- Subjects
[SDV]Life Sciences [q-bio] ,Central nervous system ,Genetic Vectors ,Gene delivery ,Biology ,Serogroup ,Applied Microbiology and Biotechnology ,Virus ,Intracardiac injection ,law.invention ,Rats, Sprague-Dawley ,03 medical and health sciences ,Transduction (genetics) ,0302 clinical medicine ,law ,intracardiac injection ,Genetics ,medicine ,Animals ,Humans ,rat ,Genetics (clinical) ,Tropism ,030304 developmental biology ,Pharmacology ,0303 health sciences ,animal model ,Myocardium ,Gene Transfer Techniques ,AAV ,Transfection ,Genetic Therapy ,Dependovirus ,Virology ,3. Good health ,Rats ,[SDV] Life Sciences [q-bio] ,medicine.anatomical_structure ,HEK293 Cells ,Blood-Brain Barrier ,Recombinant DNA ,cardiovascular system ,Molecular Medicine ,CNS ,030217 neurology & neurosurgery - Abstract
International audience; AbstractRecombinant 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.
- Published
- 2018