Marrow transplantation in the treatment of murine hereditary diseases

The prospects for analysing the curative potential of mutant stem cells following transfection with wild type genes is severely limited by the number of available animal models that fulfil all the requirements. Appropriate models must have the following characteristics: (1) the wild type stem cells are capable of providing a long-term cure of the mutant; (2) the primary product of the mutated gene is known; (3) the wild type allele is cloned; and (4) the normal gene is capable of being reinserted into mutant bone marrow stem cells where it continues to function when the cells are returned to the mutant environment. My object in this paper is to review stem cell transplantation in Mus musculus with hereditary anomalies, thus identifying the mutant mice that fulfil the first criterion and are potential models for future gene transfection studies.