Attempt at Enzyme Replacement by Fetal Liver Transplantation in Fabry's Disease

“…But these treatments have not achieved stable clinical effects. Other attempts were made, including transplantation of kidney (Clarke et al 1972) and fetal liver (Touraine et al 1979). Long-term observation is necessary for deciding the feasibility of these methods.…”

Section: Discussionmentioning

confidence: 99%

“…To date, infusions of the purified enzyme (Brady et al 1973;Tsuji et al 1994) and plasma exchange (Pyeritz et al 1980) have been used as therapies for this disease, but, unfortunately, stable clinical effects have not been achieved. Other types of treatment have been attempted, including kidney transplantation (Clarke et al 1972) and fetal liver transplantation (Touraine et al 1979). Long-term observation is necessary for evaluating these treatments.…”

Section: Introductionmentioning

confidence: 99%

Enzymatic corrections for cells derived from Fabry disease patients by a recombinant adenovirus vector

et al. 2000

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Fabry disease is an X-linked inherited metabolic disorder caused by a deficiency of α-galactosidase (α-gal), resulting in the accumulation of ceramide trihexoside (CTH) in body fluids and in many organs and tissues. We constructed a recombinant adenovirus with a human α-gal cDNA (AxCAG α-gal), and transfected this vector to skin fibroblasts from Fabry patients. Transfected cells expressed high amounts of α-gal in their cytoplasm, and a high level of α-gal activity was detected in the medium. The accumulated CTH in the fibroblasts disappeared 3 days after infection. The secreted α-gal also eliminated the accumulated CTH from uninfected patient's cells. The enzyme may be taken up through mannose-6-phosphate receptors, as the addition of mannose-6-phosphate to the medium completely inhibited the uptake of the enzyme. The infected cells continued to express α-gal for more than 10 days. These results suggest that AxCAG α-gal could be used as enzyme replacement gene therapy for Fabry disease.

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“…These observations show that the fetal liver effect is not limited to the L1210 system and they also lend further emphasis to the relative ease with which non specific tumour defenses can be modulated. Accord ingly, some caution may be advised in attempts to use fetal liver for clinical therapeutic purposes such as haemopoietic reconstitution, enzyme replacement [23] or cellular therapy ('Frischzellentherapie').…”

Section: Fetal Liver Cellsmentioning

confidence: 99%

Inhibition by Lymph Node Cells and Promotion by Fetal Hepatocytes of Murine Tumours without or with Chemotherapy

Floersheim

Torhorst²

1981

Oncology

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The growth of a syngeneic Moloney lymphoma in CBA mice was inhibited by the injection of lymph node cells from normal CBA donors. This cytotherapeutic effect was also seen after chemotherapy with cyclophosphamide (CY) or dimethylmyleran (DMM). Two other murine tumours, the Meth A sarcoma in Balb/c mice and the L1210 leukemia in BDF₁ mice, did not respond to treatment with normal syngeneic cells. In the L1210 system, tumour-promoting effects of fetal liver cells were demonstrated. The tumour inhibitory effects of normal lymph node cells against the Moloney lymphoma may be a virus-dependent phenomenon mediated through interferon and ‘natural killer’ cells. The experiments also showed the sensitivity of the Moloney lymphoma and the Meth A sarcoma to CY and DMM and the responsiveness of the L1210 leukemia to A AFC.

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Attempt at Enzyme Replacement by Fetal Liver Transplantation in Fabry's Disease

Cited by 13 publications

References 8 publications

A19-Year-Old Female with Fever, Acroparesthesia, and Progressive Deterioration of Renal Function

A19-Year-Old Female with Fever, Acroparesthesia, and Progressive Deterioration of Renal Function

Enzymatic corrections for cells derived from Fabry disease patients by a recombinant adenovirus vector

Inhibition by Lymph Node Cells and Promotion by Fetal Hepatocytes of Murine Tumours without or with Chemotherapy

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