Characterization of Recombinant Adeno-Associated Viral Transduction and Safety Profiles in Cardiomyocytes.
Cell Physiol Biochem
; 48(5): 1894-1900, 2018.
Article
em En
| MEDLINE
| ID: mdl-30092576
BACKGROUND/AIMS: Cardiovascular diseases (CVD) are the leading causes for human mortality. However, the effective treatment for these diseases are still lacking. Currently, gene therapy could be a potential way for efficiently treating heart diseases. The aim of our study is to analyze the transduction efficacy and safety profile of recombinant adeno associated virus (AAV) serotype 9 for cardiomyocytes in vivo and in vitro. METHODS: We produced rAAV serotype 9 expressing enhanced green fluorescence protein (EGFP) driven by a cardiac troponin T (cTNT) promoter, and characterized its transduction efficiency in primary cultured cardiomyocytes in vitro, and in wild-type mouse heart tissue in vivo. RESULTS: Our data showed that rAAV9 efficiently transduced mouse cardiomyocytes in vitro. Following intravenous injection, rAAV9 could efficiently and safely transduce cardiomyocytes that are involved in heart diseases. CONCLUSION: Our findings suggested that rAAV9 can efficiently and safely transduce cardiomyocytes in vitro and/or in vivo. The rAAV9 serotype vector could constitute a powerful toolbox for future gene therapy of heart diseases.
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Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transdução Genética
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Dependovirus
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Vetores Genéticos
Tipo de estudo:
Risk_factors_studies
Limite:
Animals
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Humans
Idioma:
En
Revista:
Cell Physiol Biochem
Assunto da revista:
BIOQUIMICA
/
FARMACOLOGIA
Ano de publicação:
2018
Tipo de documento:
Article
País de afiliação:
China