Bone marrow transplantation for correction of enzyme deficiency disease.

Abstract

Mutant acatalasemic mice provide a prototype of congenital enzyme deficiency disease. Normal blood catalase levels were achieved permanently in congenitally acatalasemic mice by transplantation of bone marrow cells from congeneic normal catalasemic mice using relatively small numbers of cells following whole body irradiation. The increase in blood catalase activity was physiologically effective as demonstrated by the protection of the previously acatalasemic mice against the otherwise lethal effects of hydrogen peroxide injections. Bone marrow transplantation has the potential to provide a continuous source of some enzymes and may be applicable as treatment for certain congenital enzyme deficiency diseases.

• Authors: C Hong, D E Sutherland, A J Matas, J S Najarian
• Journal: Transplantation proceedings (Transplant Proc) Vol. 11 Issue 1 Pg. 498-503 (Mar 1979) ISSN: 0041-1345 [Print] United States
• PMID: 377685 (Publication Type: Journal Article, Research Support, U.S. Gov't, P.H.S.)

Chemical References

• Catalase

Topics

• Acatalasia
• Animals
• Bone Marrow (radiation effects)
• Bone Marrow Transplantation
• Catalase (blood)
• Disease Models, Animal
• Mice
• Mice, Mutant Strains
• Radiation Chimera
• Transplantation, Homologous