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LAMP2 gene therapy for cardiomyopathy and liver dysfunction in Danon disease

DISEASE CATEGORY: Cardiovascular INDICATION: Cardiomyopathy Adeno-associated viral vector (AAV)-mediated overexpression of LAMP2 could treat Danon disease, a cardiomyopathy caused by loss-of-function LAMP2 point mutations. Danon disease causes cardiac and skeletal muscle weakness and causes multi-organ...

Complement component C1q as a hepatitis-associated liver cancer target

DISEASE CATEGORY: Hepatic; cancer INDICATION: Hepatitis; liver cancer Inhibiting the complement component C1q could treat hepatocellular carcinoma (HCC) associated with chronic hepatitis. C1q mRNA levels in the noncancerous liver tissue from patients with HBV- or...

Thioredoxin reducing enzyme inhibitors for solid tumors

DISEASE CATEGORY: Cancer INDICATION: Colorectal cancer; head and neck cancer; lung cancer A Karolinska Institute team has identified compounds that inhibit TXNRD1, a thioredoxin reductase, that could treat colorectal, hypopharyngeal and lung cancers. TXNRD1 regenerates...

YAP1 activation for recovery after spinal cord injury

DISEASE CATEGORY: Neurology INDICATION: Spinal cord injury (SCI) Activating YAP1 via inhibition of MSP and STK3 could treat SCI. In the Hippo-YAP pathway that regulates cell growth and survival, MSP and STK3 signaling phosphorylate and...

Disrupting hypoxia signaling to treat a muscular dystrophy

DISEASE CATEGORY: Musculoskeletal INDICATION: Muscular dystrophy The PI3K-mTOR pathway inhibitors could treat facioscapulohumeral muscular dystrophy (FSHD), a progressive muscle wasting disorder caused by inappropriate DUX4 expression, by disrupting the hypoxia signaling pathway. In a DUX4-expressing...

Blocking necroptosis to treat inflammatory bowel disease

DISEASE CATEGORY: Autoimmune disease INDICATION: Inflammatory bowel disease (IBD) Inhibiting necroptosis by targeting RIPK3, MLKL or ZBP1 could treat IBD associated with deficiencies in the epigenetic protein SETDB1. Bowel tissue from IBD patients had lower...

The lipids LXA4 and RvD1 as treatment for E. coli pneumonia

DISEASE CATEGORY: Infectious disease INDICATION: Escherichia; pneumonia The lipids LXA4 and RvD1, ligands of the membrane receptor FPRL1, could treat E. coli pneumonia by promoting antibacterial functions in neutrophils. The release of neutrophil elastase, induced...

A low-density lipoprotein receptor identified as tauopathy target

DISEASE CATEGORY: Neurology INDICATION: Neurology Inhibiting the low-density lipoprotein receptor LRP1 could treat tauopathies such as Alzheimer’s disease, frontotemporal dementia and chronic traumatic encephalopathy. In a human neuroglioma cell line, CRISPR-mediated inhibition of LRP1 blocked...

Apoptosis metabolites for treating arthritis, lung graft rejection

DISEASE CATEGORY: Autoimmune disease; transplant INDICATION: Arthritis; graft rejection Two cocktails of metabolites released through PANX1, a membrane channel activated during apoptosis, could treat inflammatory arthritis and lung graft rejection. The cocktails are either a...

Heparan sulfate for prevention of acetaminophen-induced acute liver failure

DISEASE CATEGORY: Hepatic INDICATION: Liver failure Inhibiting HMGB1, a DNA-binding protein, with a polysaccharide composed of 18 heparan sulfate units could prevent acute liver failure due to acetaminophen overdose. Patients with acetaminophen-induced acute liver failure...

Inhibiting an NMDA receptor to reduce lupus neurological symptoms

DISEASE CATEGORY: Autoimmune disease INDICATION: Lupus Antagonists of the NMDA receptor GRIN2A could treat systemic lupus erythematosus (SLE), an autoimmune disease that can cause neuropsychiatric symptoms including memory impairment. Cell-based assays revealed that a patient-derived...

Inhibiting endocytosis to enhance cetuximab, trastuzumab and Bavencio antitumor efficacy

DISEASE CATEGORY: Cancer INDICATION: Breast cancer; colorectal cancer; skin cancer Blocking endocytosis with DMN inhibitors could enhance the efficacy of cetuximab, trastuzumab and Bavencio avelumab against skin, breast and colorectal cancers. The mAbs target EGFR,...

Nanoparticle delivery of bee venom peptide to induce antigen release from tumors

DISEASE CATEGORY: Cancer INDICATION: Melanoma Lipid nanoparticles loaded with melittin, a bee venom peptide that induces tumor necrosis or apoptosis by disrupting cell membranes, could promote systemic antitumor responses by promoting in situ release of...

Depletion of the mitochondrial chaperone HspA9 for BRAF-mutant melanoma

DISEASE CATEGORY: Cancer INDICATION: Melanoma Reducing production of the mitochondrial chaperone HspA9 could treat BRAF-mutant melanoma. In melanoma patients, high tumor levels of HspA9 mRNA were associated with poor survival. In two human melanoma cell...

Treating pain by knocking out the ion channel TMEM120A

DISEASE CATEGORY: Neurology INDICATION: Pain Nociceptor-specific inhibition of the ion channel TMEM120A could treat pain. In cultured mouse neurons, transfection of an siRNA against TMEM120A reduced mechanically evoked currents in nociceptors. In mice, knockout or...