Encapsulation Cell Therapy for Mucopolysaccharidosis Type VII Using Genetically Engineered Immortalized Human Amniotic Epithelial Cells

Nakama, Hideyuki; Ohsugi, Kojune; Otsuki, Taisuke; Date, Isao; Kosuga, Motomichi; Okuyama, Torayuki; Sakuragawa, Norio

doi:10.1620/tjem.209.23

Cited by 20 publications

(11 citation statements)

References 32 publications

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“…These cells can also function as transgene carriers in cell-mediated gene therapy. Promising results in this area have been obtained by in vivo studies using animal models (Kosuga et al, 2001;Liu et al, 2009;Meng et al, 2008;Nakama et al, 2006;Takahashi et al, 2001). Initial experiments show that cells derived from human amniotic fluid can be transduced by viral vectors as well, or used in cell transplantation studies (Grisafi et al, 2008;Pan et al, 2009).…”

Section: Amnion-derived Stem Cellsmentioning

confidence: 99%

“…AECs are also used in cell-mediated gene therapy experiments, combining the beneficial effects of AECs themselves with the introduction of therapeutic gene (Nakama et al, 2006). Cultured AECs were shown to secrete albumin; after transfection with LacZ gene and transplantation into SCID mice, these cells were found to integrate into the liver .…”

Section: Amniotic Epithelial Cellsmentioning

confidence: 99%

See 1 more Smart Citation

On the origin of amniotic stem cells: of mice and men

Dobreva¹,

Pereira²,

Deprest³

et al. 2010

Int. J. Dev. Biol.

135

139

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A common characteristic of mammals is the development of extraembryonic supporting tissues and organs that are required for embryonic implantation, survival and development in utero. The amnion is the innermost extraembryonic membrane that eventually surrounds the fetus of amniotes, and contains the amniotic fluid. Next to its function in in utero development, the amnion has been shown to have an important potential for clinical applications. It is mainly used as a dressing to stimulate healing in skin and ocular wounds. Moreover, cells derived from the amniotic membrane and amniotic fluid have been reported to possess stem cell features, like pluripotent differentiation ability. Little is known about the early development of this membrane in humans. The mouse is a powerful genetic model organism that can be used to address the dynamics and the developmental origin of amnion and amnion-derived stem cells. Here, we discuss some fundamental differences in amnion development in the disc-shaped primate embryo and in the cup-shaped mouse embryo. We emphasize the consequences that this may have on the derivation of amniotic "stem" cells. After revision of the different isolation procedures of amniotic (fluid) derived "stem" cells from rodents, we reveal striking differences in the sources used to derive these cells across studies. The profound differences in the development of the extraembryonic membranes and cavities between primates and rodents may result in comparing cell types of different developmental origins, eventually leading to missinterpretations.

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Section: Amnion-derived Stem Cellsmentioning

confidence: 99%

Section: Amniotic Epithelial Cellsmentioning

confidence: 99%

On the origin of amniotic stem cells: of mice and men

Dobreva¹,

Pereira²,

Deprest³

et al. 2010

Int. J. Dev. Biol.

135

139

View full text Add to dashboard Cite

show abstract

“…The VEGFexpressing CHO cells significantly increased angiogenesis resulting in improvement of heart function [23]. Other approaches using encapsulated, genetically modified cells are the expression of b-glucuronidase in epithelial cells treating mucopolysaccharidosis type VII [24], the expression of erythropoietin in myoblast cells [25,26] or the expression of IL-6 in CHO cells, inhibiting tumor progression [27]. Some cell therapy systems are still in clinical phase trials.…”

Section: Stem Cells Used For Cell Therapy Approachesmentioning

confidence: 99%

Use of Encapsulated Stem Cells to Overcome the Bottleneck of Cell Availability for Cell Therapy Approaches

Freimark¹,

Pino-Grace²,

Pöhl³

et al. 2010

Transfus Med Hemother

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Nowadays cell-based therapy is rarely in clinical practice because of the limited availability of appropriate cells. To apply cells therapeutically, they must not cause any immune response wherefore mainly autologous cells have been used up to now. The amount of vital cells in patients is limited, and under certain circumstances in highly degenerated tissues no vital cells are left. Moreover, the extraction of these cells is connected with additional surgery; also the expansion in vitro is difficult. Other approaches avoid these problems by using allo-or even xenogenic cells. These cells are more stable concerning their therapeutic behavior and can be produced in stock. To prevent an immune response caused by these cells, cell encapsulation (e.g. with alginate) can be performed. Certain studies showed that encapsulated allo-and xenogenic cells achieve promising results in treatment of several diseases. For such cell therapy approaches, stem cells, particularly mesenchymal stem cells, are an interesting cell source. This review deals on the one hand with the use of encapsulated cells, especially stem cells, in cell therapy and on the other hand with bioreactor systems for the expansion and differentiation of mesenchymal stem cells in reproducible and sufficient amounts for potential clinical use.

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“…The underlining potential advantages include 1) that transplanted cells do not have physical contact with host cells, thus protecting cells from immune mediators; 2) and that the risk of uncontrolled proliferation of transplanted cells may be put under check. Significant β-glucuronidase production and release has been observed in MPS VII brain after transplantation of encapsulated enzyme-secreting fibroblasts [95], or immortalized human amniotic epithelial cells [96] into adult mice. Direct in vivo producer cell-mediated gene transfer was also investigated by transplanting encapsulated retroviral packaging cells into murine MPS VII brain for continuous vector production and gene transfer for up to 6 weeks [97].…”

Section: Immunology Of Intracranial Deliverymentioning

confidence: 99%

Cell-and Gene-based Therapeutic Approaches for Neurological Deficits in Mucopolysaccharidoses

Pan¹

2011

CPB

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Mucopolysaccharidoses (MPS) are a group of lysosomal storage diseases that are resulted from abnormal accumulation of glycosaminoglycans. Among the progressive multi-organ abnormalities often associated with MPS diseases, the deterioration of central nervous system (CNS) is the most challenging manifestations to be tackled, due to the impermeability of the blood-brain-barrier (BBB). Evolved with recent development in stem cell biotechnology and gene therapy, several novel experimental approaches have been investigated in animal models. In this review, we will address different approaches attempting to bypass the BBB for neuropathic MPS treatment using cell-and gene-based therapies. Several neurological findings in CNS pathophysiology emerged with therapeutic investigation will also be discussed.

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Encapsulation Cell Therapy for Mucopolysaccharidosis Type VII Using Genetically Engineered Immortalized Human Amniotic Epithelial Cells

Cited by 20 publications

References 32 publications

On the origin of amniotic stem cells: of mice and men

On the origin of amniotic stem cells: of mice and men

Use of Encapsulated Stem Cells to Overcome the Bottleneck of Cell Availability for Cell Therapy Approaches

Cell-and Gene-based Therapeutic Approaches for Neurological Deficits in Mucopolysaccharidoses

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