Immunotherapy for lymphangioleiomyomatosis and tuberous sclerosis: progress and future directions

HJ Liu, VP Krymskaya, EP Henske - Chest, 2019 - Elsevier
Pulmonary lymphangioleiomyomatosis (LAM) is a rare genetic multisystem disease
characterized by the nodular proliferation of smooth muscle-like LAM cells, progressive
cystic changes of the lung, lymphatic abnormalities, and renal angiomyolipomas (AMLs).
LAM can arise sporadically or in women with the autosomal dominant disorder, tuberous
sclerosis complex (TSC), in which hamartomatous tumors of brain, heart, skin, kidney, and
lung are found. LAM and TSC are caused by mutations in the TSC1 or TSC2 tumor …