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Lo, Bernice; Fritz, Jill M.; Su, Helen C.; Uzel, Gulbu; Jordan, Michael B.; Lenardo, Michael J.
Blood, 08/2016, Volume: 128, Issue: 8Journal Article
CTLA-4 is a critical inhibitory “checkpoint” molecule of immune activation. Several recent reports have described patients with immune dysregulation and lymphoproliferative disease resulting from 2 different genetic diseases that directly or indirectly cause CTLA-4 deficiency. Numerous articles have also been published describing CTLA-4 blockade in cancer immunotherapy and its side effects, which are ultimately the consequence of treatment-induced CTLA-4 deficiency. Here, we review these 2 diseases and CTLA-4 blockade therapy, emphasizing the crucial role of CTLA-4 in immune checkpoint regulation.
