SMA is an inherited disease that leads to loss of motor function and ambulation and a
reduced life expectancy. We have been working to develop orally administrated …

Spinal muscular atrophy (SMA) is a motor neuron disease, typically resulting from loss-of-
function mutations in the survival motor neuron 1 (SMN1) gene. Nusinersen/SPINRAZA, a …

Epithelial–mesenchymal transition (EMT) is a critical event in metastasis of colorectal cancer
(CRC). Rho/ROCKs signaling has a pivotal role in orchestrating actin cytoskeleton, leading …

Aims Aggressive meningioma remains incurable with neither chemo‐nor targeted therapies
proven effective, largely due to unidentified genetic alterations and/or aberrant oncogenic …

Background Triple-negative breast cancer (TNBC) is an aggressive disease. Recent studies
have identified genome instability-derived genes for patient outcomes. However, most of the …

B-RafV600E oncogene mutation occurs most commonly in papillary thyroid carcinoma
(PTC) and is associated with tumor initiation. However, a genetic modification by B …

foxm1 is a master regulator of the cell cycle, contributing to cell proliferation. Recent data
have shown that this transcription factor also modulates gene networks associated with …

Targeting RNA drastically expands our target space to therapeutically modulate numerous
cellular processes implicated in human diseases. Of particular interest, drugging pre-mRNA …

Background: Docetaxel used for first-line treatment of advanced prostate cancer (PCa) is
only marginally effective. We previously showed, using the LTL-313H subrenal capsule …

BACKGROUND AND OBJECTIVES: Glioma accounts for most of primary malignant brain
tumors and usually results in poor survival. However, the key signaling networks regulating …