Adenosine deaminase (ADA) deficiency in humans leads to a combined immunodeficiency.
The mechanisms involved in the lymphoid specificity of the disease are not fully understood
due to the inaccessibility of human tissues for detailed analysis and the absence of an
adequate animal model for the disease. We report the use of a two-stage genetic
engineering strategy to generate ADA-deficient mice that retain many features associated
with ADA deficiency in humans, including a combined immunodeficiency. Severe T and B …