Adenosine deaminase (ADA; EC) deficiency in humans is an autosomal recessive genetic
disorder that results in severe combined immunodeficiency disease. ADA-deficient mice
generated by targeted gene disruption die perinatally, preventing postnatal analysis of ADA
deficiency. We have recently rescued ADA-deficient fetuses from perinatal lethality by
expression of an ADA minigene in the placentas of ADA-deficient fetuses, thus generating
postnatal mice admissible to analysis of ADA deficiency. The minigene used also directed …