Long-term enzyme replacement therapy in beta-glucuronidase--deficient mice by allogeneic bone marrow transplantation

Journal Article · Tue Jun 01 00:00:00 EDT 1982 · J. Lab. Clin. Med.; (United States)

OSTI ID:7055241

Yatziv, S; Weiss, L; Morecki, S; Fuks, Z; Slavin, S

Enzyme replacement therapy was successfully accomplished in beta-Glu-deficient C3H/HeJ mice after transplantation of BM cells obtained from normal BALB/c donors. Marrow recipients were prepared for transplantation by fractionated TLI. Enzyme activity increased from 20.5 +/- 7.0 nmol/mg of protein per hour to 180 +/- 30.2 in the liver (p less than 0.001) and from 8.2 +/- 2.0 to 17.5 +/- 5.0 nmol/ml/hr in the plasma (p less than 0.05) at 50 days after marrow infusion. Normal enzyme activity was maintained in treated mice for at least 100 days after marrow transplantation, as documented by repeated liver biopsies and examination of plasma samples. The marrow donors and the recipients were fully histoincompatible. Both immunologic rejection of the marrow allograft and GVHD were prevented by the prior conditioning of the recipients with TLI, resulting in bilateral transplantation tolerance of host vs. graft and graft vs. host. The data suggest that allogeneic BM transplantation may provide a possible therapeutic approach for certain enzyme deficiency syndromes.

• Research Organization: Hadassah University Hospital, Jerusalem, Israel
• OSTI ID: 7055241
• Journal Information: J. Lab. Clin. Med.; (United States), Vol. 99:6
• Country of Publication: United States
• Language: English

Related Subjects

62 RADIOLOGY AND NUCLEAR MEDICINE
63 RADIATION, THERMAL, AND OTHER ENVIRON. POLLUTANT EFFECTS ON LIVING ORGS. AND BIOL. MAT.
BONE MARROW CELLS
TRANSPLANTS
LYMPHATIC SYSTEM
BIOLOGICAL RADIATION EFFECTS
METABOLIC DISEASES
RADIOTHERAPY
ENZYME ACTIVITY
FRACTIONATED IRRADIATION
GRAFT-HOST REACTION
IMMUNOSUPPRESSION
MICE
WHOLE-BODY IRRADIATION
ANIMAL CELLS
ANIMALS
BIOLOGICAL EFFECTS
CONNECTIVE TISSUE CELLS
DISEASES
EXTERNAL IRRADIATION
IRRADIATION
MAMMALS
MEDICINE
NUCLEAR MEDICINE
RADIATION EFFECTS
RADIOLOGY
RODENTS
SOMATIC CELLS
THERAPY
VERTEBRATES
550603* - Medicine- External Radiation in Therapy- (1980-)
560152 - Radiation Effects on Animals- Animals