Ex Vivo Lentivirus Transduction and Immediate Transplantation of Uncultured Hepatocytes for Treating Hyperbilirubinemic Gunn Rat

Nguyen, Tuan Huy; Birraux, Jacques; Wildhaber, Barbara E.; Myara, Anne; Trivin, F; Coultre, Claude Pierrette Le; Trono, Didier; Chardot, Christophe

doi:10.1097/01.tp.0000234675.56598.35

Cited by 31 publications

(10 citation statements)

References 49 publications

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“…In this study, the results have shown a lack of toxicity and persistent gene expression lasting at least four months following gene therapy. Successful reports of human hepatocyte genetic correction, using lentiviral vectors, followed by transplantation in the Gunn rat [94] [10,92] and nonhuman primates [82] have been published. These findings confirm the potential feasibility of gene therapy by using lentiviral vectors to treat liver-based inborn errors of metabolism, which are a prerequisite to clinical applications (for review see [93]).…”

Section: Human Ips Cell Therapies For Hereditary Metabolic Liver Disementioning

confidence: 98%

Induced pluripotent stem cells: A new era for hepatology

Asgari

Pournasr

Salekdeh

et al. 2010

Journal of Hepatology

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Stem cell transplantation has been proposed as an attractive alternative approach to restore liver mass and function. Recent progress has been reported on the generation of induced pluripotent stem (iPS) cells from somatic cells. Human-iPS cells can be differentiated towards the hepatic lineage which presents possibilities for improving research on diseases, drug development, tissue engineering, the development of bio-artificial livers, and a foundation for producing autologous cell therapies that would avoid immune rejection and enable correction of gene defects prior to cell transplantation. This focused review will discuss how human iPS cell advances are likely to have an impact on hepatology.

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Section: Human Ips Cell Therapies For Hereditary Metabolic Liver Disementioning

confidence: 98%

Induced pluripotent stem cells: A new era for hepatology

Asgari

Pournasr

Salekdeh

et al. 2010

Journal of Hepatology

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“…This transduction was maintained for up to 4 months after injection in the study animals [70] . In addition, the lentiviral vectors were able to transduce a wide range of post-mitotic organs such as muscle [71] , retina [72] , neurons [73] and liver [74] .…”

Section: Lentivirusmentioning

confidence: 99%

Exploitation of stem cell homing for gene delivery

Penn¹,

Khalil

2007

Expert Opinion on Biological Therapy

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Stem cells have been the focus of numerous investigations to treat diseases as far ranging as diabetes, chronic heart failure and multiple sclerosis over the past decade. The process of stem-cell-based repair of acute injury involves homing and engrafting of the stem cell of interest to the site of injury followed by either differentiation of the stem cell to indigenous end-organ cells or liberation of paracrine factors that lead to preservation and/or optimization of organ function. Recognition of the ability of stem cells to home to sites of acute injury suggests that, if appropriately defined and harnessed, stem cell homing could serve as a means of local drug delivery through the infusion of genetically engineering stem cells that secrete gene products of interest. The authors have recently demonstrated the use of this approach in preclinical studies of acute myocardial function. In addition, the use of engineered cells that home to appropriate niches have been used to correct genetic deficiency states (i.e., severe combined immunodeficiency, diabetes mellitus) in patients with otherwise chronic debilitating diseases. This review focuses on exploiting stem cell homing for gene transfer and on the state of the art and the challenges that face the field.

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“…65 Titers of LV preparations were thus calculated as the number of transducing units per ml. The same qPCR assay was used to determine copy numbers of integrated LV in stably transduced Huh7 and HepG2.2.15 cells.…”

Section: Cell Culturementioning

confidence: 99%

Sustained inhibition of hepatitis B virus replication in vivo using RNAi-activating lentiviruses

et al. 2014

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Chronic infection with hepatitis B virus (HBV) puts individuals at high risk for complicating cirrhosis and liver cancer, but available treatment to counter the virus rarely eliminates infection. Although harnessing RNA interference (RNAi) to silence HBV genes has shown the potential, achieving efficient and durable silencing of viral genes remains an important goal. Here we report on the propagation of lentiviral vectors (LVs) that successfully deliver HBV-targeting RNAi activators to liver cells. Mono- and tricistronic artificial primary microRNAs (pri-miRs) derived from pri-miR-31, placed under transcriptional control of the liver-specific modified murine transthyretin (mTTR) promoter, caused efficient inhibition of HBV replication markers. The tricistronic cassette was capable of silencing a mutant viral target and the effects were observed without disrupting the function of an endogenous miR (miR-16). The mTTR promoter stably expressed a reporter transgene in mouse livers over a study period of 1 year. Good silencing of HBV genes, without evidence of toxicity, was demonstrated following intravenous injection of LVs into neonatal HBV transgenic mice. Collectively, these data indicate that LVs may achieve sustained inhibition of HBV replication that is appealing for their therapeutic use.

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Ex Vivo Lentivirus Transduction and Immediate Transplantation of Uncultured Hepatocytes for Treating Hyperbilirubinemic Gunn Rat

Cited by 31 publications

References 49 publications

Induced pluripotent stem cells: A new era for hepatology

Induced pluripotent stem cells: A new era for hepatology

Exploitation of stem cell homing for gene delivery

Sustained inhibition of hepatitis B virus replication in vivo using RNAi-activating lentiviruses

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