Generation of inactivated IL2RG and RAG1 monkeys with severe combined immunodeficiency using base editing.

ABSTRACT: Severe combined immunodeficiency (SCID) encompasses a range of inherited disorders that lead to a profound deterioration of the immune system. Among the pivotal genes associated with SCID, RAG1 and IL2RG play crucial roles. IL2RG is essential for the development, differentiation, and functioning of T, B, and NK cells, while RAG1 critically contributes to adaptive immunity by facilitating V(D)J recombination during the maturation of lymphocytes. Animal models carrying mutations in these genes exhibit notable deficiencies in their immune systems. Non-human primates (NHPs) are exceptionally well-suited models for biomedical research due to their genetic and physiological similarities to humans. Cytosine base editors (CBEs) serve as powerful tools for precisely and effectively modifying single

SUBMITTER: Zheng X 

PROVIDER: S-EPMC10475462 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature