In preclinical studies, dual-targeted agents that directly inhibit both PI3K and mTOR (e.g., PI-103, NVP-BEZ235) have shown efficacy in Ph+ pre-B cell acute leukemia (Kharas et al., 2008), chronic lymphocytic leukemia (CLL; Niedermeier et al., 2009), various B cell lymphomas (Bhatt et al., 2010; Bhende et al., 2010), and MM (Baumann et al., 2009). The gene discussed is MTOR; the disease is Miyoshi myopathy.