Efficient and long-term intracardiac gene transfer in δ-sarcoglycan-deficiency hamster by adeno-associated virus-2 vectors

Li, J; Wang, D; Shen, Qian; Chen, Z; Zhu, Tong; Xiao, Xianmin

doi:10.1038/sj.gt.3302078

Cited by 58 publications

(34 citation statements)

References 44 publications

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“…Compared with the reports in which various strategies of gene transduction to the donor heart using adenovirus and AAV (Table 1), (1,25,26) the efficiency of transduction in this study was excellent. rAAV9-mediated gene transfer to the transplanted heart using a cold perfusion system seems to be a clinically relevant gene therapy approach.…”

Section: Discussioncontrasting

confidence: 47%

Efficient and Durable Gene Transfer to Transplanted Heart Using Adeno-associated Virus 9 Vector

Miyagi

Rao

Ricci

et al. 2008

The Journal of Heart and Lung Transplantation

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Section: Discussioncontrasting

confidence: 47%

Efficient and Durable Gene Transfer to Transplanted Heart Using Adeno-associated Virus 9 Vector

Miyagi

Rao

Ricci

et al. 2008

The Journal of Heart and Lung Transplantation

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“…In histological examination, we have reevaluated rAAV-LacZ-transferred hamster myocardium, searching for β-gal-positive staining in the coronary vascular bed and in the perivascular interstitial area, and we observed none. Lack of vascular gene expression after the transcoronary perfusion of rAAV vectors was independently reported by Xiao's group (37). Thus, we speculate that S16EPLN expressed in cardiomyocytes exerted beneficial effects in the failing heart.…”

Section: Figurementioning

confidence: 84%

Chronic phospholamban inhibition prevents progressive cardiac dysfunction and pathological remodeling after infarction in rats

Iwanaga¹,

Hoshijima²,

Gu³

et al. 2004

J. Clin. Invest.

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Ablation or inhibition of phospholamban (PLN) has favorable effects in several genetic murine dilated cardiomyopathies, and we showed previously that a pseudophosphorylated form of PLN mutant (S16EPLN) successfully prevented progressive heart failure in cardiomyopathic hamsters. In this study, the effects of PLN inhibition were examined in rats with heart failure after myocardial infarction (MI), a model of acquired disease. S16EPLN was delivered into failing hearts 5 weeks after MI by transcoronary gene transfer using a recombinant adeno-associated virus (rAAV) vector. In treated (MI-S16EPLN, n = 16) and control (MI-saline, n = 18) groups, infarct sizes were closely matched and the left ventricle was similarly depressed and dilated before gene transfer. At 2 and 6 months after gene transfer, MI-S16EPLN rats showed an increase in left ventricular (LV) ejection fraction and a much smaller rise in LV end-diastolic volume, compared with progressive deterioration of LV size and function in MI-saline rats. Hemodynamic measurements at 6 months showed lower LV end-diastolic pressures, with enhanced LV function (contractility and relaxation), lowered LV mass and myocyte size, and less fibrosis in MI-S16EPLN rats. Thus, PLN inhibition by in vivo rAAV gene transfer is an effective strategy for the chronic treatment of an acquired form of established heart failure.

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“…It has been extensively demonstrated that rAAV can successfully infect and transduce a broad variety of cell and tissue types, such as brain, liver, muscle, etc [During, MJ et al, 1998;Song, S et al, 1998;Ye, X et al, 1999;Acland, GM et al, 2001; and has the ability to infect both dividing and quiescent cells [Lewis, PF and Emerman, M. 1994;Alexander, IE et al, 1996]. Not only can rAAV infect a broad spectrum of cell types, long term gene expression greater than 1.5 years has also been demonstrated in animal models including canine, murine and hamster [McCown, TJ et al, 1996;Monahan, PE et al, 1998;Herzog, RW et al, 1999;Snyder, RO et al, 1999;Cottard, V et al, 2000;Song, S et al, 2001;Asfour, B et al, 2002;Li. J et al, 2003].…”

Section: Introductionmentioning

confidence: 99%

AAV Hybrid Serotypes: Improved Vectors for Gene Delivery

Choi¹,

McCarty²,

Samulski³

2005

CGT

165

128

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In recent years, significant efforts have been made on studying and engineering adeno-associated virus (AAV) capsid, in order to increase efficiency in targeting specific cell types that are nonpermissive to wild type (wt) viruses and to improve efficacy in infecting only the cell type of interest. With our previous knowledge of the viral properties of the naturally occurring serotypes and the elucidation of their capsid structures, we can now generate capsid mutants, or hybrid serotypes, by various methods and strategies. In this review, we summarize the studies performed on AAV retargeting, and categorize the available hybrid serotypes to date, based on the type of modification: 1) transcapsidation, 2) adsorption of bi-specific antibody to capsid surface, 3) mosaic capsid, and 4) chimeric capsid. Not only these hybrid serotypes could achieve high efficiency of gene delivery to a specific targeted cell type, which can be better-tailored for a particular clinical application, but also serve as a tool for studying AAV biology such as receptor binding, trafficking and genome delivery into the nucleus. Keywords

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Efficient and long-term intracardiac gene transfer in δ-sarcoglycan-deficiency hamster by adeno-associated virus-2 vectors

Cited by 58 publications

References 44 publications

Efficient and Durable Gene Transfer to Transplanted Heart Using Adeno-associated Virus 9 Vector

Efficient and Durable Gene Transfer to Transplanted Heart Using Adeno-associated Virus 9 Vector

Chronic phospholamban inhibition prevents progressive cardiac dysfunction and pathological remodeling after infarction in rats

AAV Hybrid Serotypes: Improved Vectors for Gene Delivery

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