Successful expression of human factor IX following repeat administration of adenoviral vector in mice.

Walter, Johannes; You, Qimin; Hagstrom, J. Nathan; Sands, Mark S.; High, Katherine A.

doi:10.1073/pnas.93.7.3056

Cited by 122 publications

(84 citation statements)

References 20 publications

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“…Administration of adenovirus vectors into the trachea, skeletal muscle, thoracic cavity or vasculature of neonatal mice or rats results in transgene expression for several weeks or months. 12,13,[18][19][20][21][22] Subsequent administration of the adenovirus vectors were tolerated and resulted in robust gene expression in some studies 19,20,23 but not others. 18 The relative success of this approach in rodents has been attributed to the relative immaturity of the immune system in the rodent at birth.…”

Section: Discussionmentioning

confidence: 99%

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Pulmonary inflammation associated with repeated, prenatal exposure to an E1, E3-deleted adenoviral vector in sheep

et al. 1999

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Fetal gene therapy may prove useful in treating diseases electively killed and 13 died in utero. The incidence of morthat manifest in the perinatal or early postnatal period.tality was higher than the р10% we have experienced with Adenoviruses effectively transfer gene expression to a varother fetal sheep studies and was not likely related to comiety of tissues but also stimulate inflammatory and immune plications arising from surgical or anesthetic procedures. responses. The purpose of this study was to test the Inflammatory and fibrotic responses were observed in the hypothesis that exposure of fetal sheep to a first generation lungs and may represent untoward long-term conseadenovirus vector encoding bacterial ␤-galactosidase, quences of in utero adenoviral gene therapy. Tolerance to Av1nBg, before the development of the immune system, is Av1nBg was not established, and repeated exposure to safe, minimizes inflammatory and immune responses and Av1nBg before birth was associated with significant patholinduces tolerance. A total of 22 fetal sheep was studied; of ogy and mortality. these, two were born with respiratory distress, seven were

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Section: Discussionmentioning

confidence: 99%

“…Studies in neonatal mice and rats have demonstrated that priming with an initial intravenous dose of adenovirus vector resulted in tolerance to subsequent administration of the same vector. 19,23 The intravenous route, which delivers …”

Section: Discussionmentioning

confidence: 99%

Pulmonary inflammation associated with repeated, prenatal exposure to an E1, E3-deleted adenoviral vector in sheep

et al. 1999

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“…29,30 This new system has the theoretical advantage ence with adenoviral vectors is mainly a function of the immunogenicity of adenoviral proteins. 23,27 However, of eliminating possible viral protein-mediated immunogenicity. Factors contributing to lack of persistence will more recent data show that the expressed transgene carried by the adenoviral vector is the major immunogen.…”

Section: Figure 4 Comparison Of Multiple Rh-leptin Daily Injections Wmentioning

confidence: 99%

Leptin gene therapy and daily protein administration: a comparative study in the ob/ob mouse

Morsy

Zhao

et al. 1998

Gene Ther

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We have compared the efficacy of daily injection of recomgiven as mean ± standard error of the mean.) Importantly, binant leptin protein (rh-leptin) with adenovirus-mediated rh-leptin and ex vivo-expressed Ad-leptin were equivalently delivery of the murine or human leptin gene (Ad-leptin) for active in a functional cell-based assay. The primary differtreatment of obesity in the obese (ob/ob) mouse model. ence in the two therapeutic approaches is the continuous We demonstrate an improved correction profile for obesity chronic secretion of leptin mediated by gene delivery, verand associated surrogate markers using the adenovirus sus the intermittent bolus delivery and rapid clearance of delivery method. Rate of weight loss and percentage satthe daily injection of rh-leptin protein. Thus, in vivo findings iety were significantly greater in the mice treated with Adsuggest that leptin effects are better achieved at lower leptin. These findings were associated with lower peak steady-state levels, a pharmacological feature attained serum leptin levels with Ad-leptin (22.9 ± 2.6 ng/ml for the here by gene therapy. These findings may have implihuman gene, and 48.9 ± 11.5 ng/ml for the murine gene) cations for the potential use of leptin in the treatment of compared to rh-leptin (385.2 ± 36.0 ng/ml). (Values are obesity.

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“…[2][3][4][5][6][7][8][9][10][11][12][13][14] Their immunogenicity has been exploited to study the possibility of fetal immune tolerance after prenatal administration. [15][16][17] However, loss of these episomal DNA vectors during subsequent cell divisions following infection severely curtails their applicability to long-term fetal gene therapy.…”

Section: Introductionmentioning

confidence: 99%

Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice

Waddington¹,

Mitrophanous

Ellard

et al. 2003

Gene Ther

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Successful expression of human factor IX following repeat administration of adenoviral vector in mice.

Abstract: ABSTRACT

Cited by 122 publications

References 20 publications

Pulmonary inflammation associated with repeated, prenatal exposure to an E1, E3-deleted adenoviral vector in sheep

Pulmonary inflammation associated with repeated, prenatal exposure to an E1, E3-deleted adenoviral vector in sheep

Leptin gene therapy and daily protein administration: a comparative study in the ob/ob mouse

Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice

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