Nonmyeloablative Matched Unrelated Donor Hematopoietic Cell Transplant for Late-Onset Omenn's with Novel RAG Mutation

Abstract

RATIONALE: Omenn syndrome is an autosomal recessive form of severe combined immunodeficiency characterized by erythematous rash, hepatosplenomegaly, lymphadenopathy, and recurrent infections due to mutations in the recombination activating genes.