distillery

CAPN3 gene therapy for limb-girdle muscular dystrophy type 2A

DISEASE CATEGORY: Musculoskeletal INDICATION: Muscular dystrophy An adeno-associated viral vector deliverying CAPN3, a calcium-dependent protease expressed at low levels in the heart and primarily expressed in skeletal muscle, could treat limb-girdle muscular dystrophy type 2A...

An inhibitor of viral latency for imaging, treating EBV-associated cancers

DISEASE CATEGORY: Cancer INDICATION: Nasopharyngeal cancer; lymphoma A peptide-based compound that inhibits EBNA1, which promotes Epstein-Barr virus latency, could be used to image and treat EBV-associated nasopharyngeal cancer and lymphoma. The compound consists of a...

Skin grafts based on CRISPR-edited iPS cells to treat epidermolysis bullosa

DISEASE CATEGORY: Dermatology INDICATION: Blistering disorder Skin grafts based on CRISPR-edited induced pluripotent stem (iPS) cells could treat recessive dystrophic epidermolysis bullosa, a blistering disorder caused by mutations in COL7A1. Generation of the full-thickness grafts...

Inhibiting the EIF4G1-EIF4E interaction to treat autism

DISEASE CATEGORY: Neurology INDICATION: Autism Disrupting the EIF4G1-EIF4E interaction could treat autism, a disorder characterized by social deficits and associated with increased dendritic spine densities and mutations in the E3 ligase complex component CUL3. In...

Raising PDLIM2 expression to treat lung cancer

DISEASE CATEGORY: Cancer INDICATION: Lung cancer Promoting the expression of PDLIM2, which has been proposed to have tumor suppressor functions, could treat lung cancer. In lung cancer patients, PDLIM2 RNA levels were lower in tumor...

SLC5A12 inhibition for rheumatoid arthritis

DISEASE CATEGORY: Autoimmune disease INDICATION: Rheumatoid arthritis (RA) Inhibiting the lactate transporter SLC5A12 could treat RA. In 87 patients with early RA, high levels of SLC5A12 RNA expression in the synovium was associated with increased...

Depleting platelets of inhibiting SOCS3 for atherosclerosis

DISEASE CATEGORY: Cardiovascular INDICATION: Atherosclerosis Platelet depletion or inhibition of SOCS3, a platelet-induced regulator of macrophage polarization, could treat atherosclerosis. Blood levels of SOCS3 RNA were higher in 61 women presenting with myocardial infarction (MI)...

Combined inhibition of EIF4A1 and glutaminase for pancreatic cancer

DISEASE CATEGORY: Cancer INDICATION: Pancreatic cancer Inhibiting the translation initiator EIF4A1 and the glutamine metabolism enzyme GLS could treat pancreatic ductal adenocarcinoma (PDAC). An EIF4A1 inhibitor tool compound lowered the viability of mouse PDAC organoids...

Gut microbe metabolites as treatments for iron overload

DISEASE CATEGORY: Hematology INDICATION: Iron overload The microbial metabolites diaminopropane and reuterin could treat iron overload by inhibiting HIF2A, which promotes iron absorption from the gut. Microbes in the gut compete with the host to...

Benzoxaborole-based CPSF3 inhibitor to treat infections

DISEASE CATEGORY: Infectious disease INDICATION: Cryptosporidium Inhibiting the Cryptosporidium CPSF3 homolog with AN3661 could treat infections by the parasite, which can be lethal in young children and immunocompromised individuals. Anacor Pharmaceuticals Inc., which Pfizer Inc....

Promoting calcium channel signaling in muscle to treat age-related atrophy

DISEASE CATEGORY: Musculoskeletal INDICATION: Muscular atrophy Increasing signaling through the embryonic isoform of the skeletal muscle-specific calcium channel CACNB1 or its downstream effector GDF5 could treat age-related muscular atrophy. Transcript levels of both proteins were...

Negative allosteric modulators of glutamate receptor subtypes mGluR2 and mGluR3

DISEASE CATEGORY: Neurology INDICATION: Depression Negative allosteric modulators of the glutamate receptor subtypes mGluR2 and mGluR3 could treat depression. Depression is characterized by reduced active coping mechanisms, intentional measures taken to lower stress, and more...

CXCR2 inhibition could overcome androgen receptor antagonist resistance in prostate cancer

DISEASE CATEGORY: Cancer INDICATION: Prostate cancer Inhibiting CXCR2 could treat androgen receptor antagonist-resistant prostate cancer. Knockout of CXCR2, which is found on treatment-resistant, androgen receptor-negative neuroendocrine tumor cells in heterogeneous prostate cancers, restored sensitivity to...

Targeting the integrated stress response to treat Down syndrome

DISEASE CATEGORY: Neurology INDICATION: Cognitive dysfunction Suppressing the integrated stress response (ISR) by activating EIF2B or inhibiting EIF2AK2 could treat Down syndrome. Levels of phosphorylation of EIF2AK2 and of its downstream target EIF2, both markers...

The proteasome-regulating kinase DYRK2 as a target in breast cancer and multiple myeloma

DISEASE CATEGORY: Cancer INDICATION: Breast cancer; multiple myeloma (MM) Inhibiting the proteasome-regulating kinase DYRK2 could treat breast cancer and MM. In patients with triple-negative breast cancer (TNBC) or MM, DYRK2 expression and protein levels were...