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Inhibiting translation initiation for androgen receptor-deficient prostate cancer

DISEASE CATEGORY: Cancer INDICATION: Prostate cancer Disrupting the eIF4F translation initiation complex formation could treat androgen receptor-deficient prostate cancers. Low expression of the translation inhibitor e1F4EBP1, which when hypophosphorylated prevents eIF4F complex formation, correlated with...

Lysosomal ceramidase ASAH1 identified as Krabbe disease target

DISEASE CATEGORY: Neurology INDICATION: Leukodystrophy A team from BioMarin Pharmaceutical Inc. and Washington University School of Medicine in St. Louis showed that inhibiting the lysosomal ceramidase ASAH1 was able to treat globoid cell leukodystrophy (Krabbe...

Novel targets induce cardiomyocyte regeneration

DISEASE CATEGORY: Cardiovascular INDICATION: Myocardial infarction (MI) Gene therapies delivering CCL24 or IGF2BP3 could restore cardiac function following MI. RNA-seq analysis identified 874 genes including CCL24 and IGF2BP3 that were upregulated in mouse heart tissue...

Inhibiting caspase-mediated pyroptosis for graft-versus-host disease

DISEASE CATEGORY: Transplant INDICATION: Graft-versus-host disease (GvHD) Inhibiting CASP4 or CASP5, the human homologs of murine pyroptosis activator CASP11, or the downstream mediators GSDMD and IL-1α could prevent GvHD in leukemia patients undergoing allogeneic hematopoietic...

Bispecific cytokine fusion for Type II diabetes

DISEASE CATEGORY: Endocrine/metabolic disease INDICATION: Diabetes A chimeric protein derived from two cytokines previously shown to modify food intake, IL-6 and CNTF, could treat Type II diabetes. The chimeric cytokine is based on IL-6, with...

SERPINB1 and CXCR6 inhibition for multiple sclerosis

DISEASE CATEGORY: Autoimmune disease INDICATION: Multiple sclerosis (MS) Blocking the leukocyte esterase inhibitor SERPINB1 or the chemokine receptor CXCR6 could help treat MS. In the experimental autoimmune encephalomyelitis (EAE) mouse model of MS, systemic knockout...

Blocking the NAD+ biosynthesis enzyme NamPRT for PPM1D-mutant tumors

DISEASE CATEGORY: Cancer INDICATION: Brain cancer; sarcoma; breast cancer Inhibiting NamPRT, a nicotinamide adenine dinucleotide (NAD+) biosynthesis enzyme, could treat PPM1D-mutant cancers including glioma, osteosarcoma and breast cancer. In tumor biopsies from glioma patients, high...

Increasing SLC8B1 expression for Alzheimer's

DISEASE CATEGORY: Neurology INDICATION: Alzheimer's disease (AD) Promoting the expression of SLC8B1, a mediator of mitochondrial calcium efflux, could treat AD. Postmortem frontal cortex samples from patients with non-familial, sporadic AD had lower levels of...

Inhibiting GPR139 for opioid withdrawal

DISEASE CATEGORY: Neurology INDICATION: Addiction Inhibiting GPR139 could treat opioid withdrawal. In a mutagenesis screen in Caenorhabditis elegans , a Scripps Research Institute team associated the C. elegans homolog of GPR139 with regulation of morphine...

DLK identified as target for Fragile X syndrome

DISEASE CATEGORY: Neurology INDICATION: Fragile X syndrome Inhibiting DLK, a mediator of neuronal injury responses, could treat Fragile X syndrome. In fruit flies with the disease, systemic DLK knockout or a tool compound DLK inhibitor...

S100A10 and SP1 identified as ALS targets

DISEASE CATEGORY: Neurology INDICATION: Amyotrophic lateral sclerosis (ALS) Inhibiting the adaptor protein S100A10 or SP1, a transcription factor that controls S100A10 expression, could treat ALS and injury-induced motor neuron degeneration. In mice with ALS, siRNA...

TAP inhibition for multiple cancers

DISEASE CATEGORY: Cancer INDICATION: B cell lymphoma; T cell lymphoma; breast cancer; pancreatic cancer Inhibiting TAP could treat B and T cell lymphoma, breast and pancreatic cancers. An siRNA targeting TAP conjugated to an oligonucleotide...

A Zika vaccine based on envelope protein dimers

DISEASE CATEGORY: Infectious disease INDICATION: Zika virus A vaccine based on Zika virus Env dimers could prevent infection with the virus. The vaccine consist of Env dimers with three disulfide bridges stably linking the subunits...

Treating Parkinson's with the resolvin RvD1

DISEASE CATEGORY: Neurology INDICATION: Parkinson's disease (PD) The resolvin RvD1, a metabolite of omega-3 fatty acids, could treat Parkinson’s disease by preventing inflammation in the brain. CSF and plasma levels of RvD1 were lower in...

SLC7A11 and mGluR5 identified as targets for hepatic alcoholic steatosis

DISEASE CATEGORY: Hepatic INDICATION: Liver disease Inhibiting SLC7A11, which excretes glutamate from cells, or the glutamate receptor mGluR5 could treat alcoholic steatosis of the liver. Patients with alcoholic fatty liver disease had elevated plasma levels...