distillery

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...

NUDT1 inhibitor-carrying nanoparticles for solid tumors

DISEASE CATEGORY: Cancer INDICATION: Breast cancer; cervical cancer; colorectal cancer; melanoma Nanoparticles delivering NUDT1 inhibitors could enhance the efficacy of photodynamic therapy in breast, cervical, colorectal and skin cancers. The therapy comprises silica cross-linked micelles...

Targeting CD36 for melanoma and colorectal cancer

DISEASE CATEGORY: Cancer INDICATION: Colorectal cancer; melanoma Inhibiting CD36, a scavenger receptor for uptake of long-chain fatty acids and oxidized low-density lipoproteins, in regulatory T cells (Tregs) could treat colorectal cancer and melanoma. In a...

The bacterial adhesion protein fimH as a cancer immunotherapy adjuvant

DISEASE CATEGORY: Cancer INDICATION: Colorectal cancer; melanoma The E. coli adhesion protein fimH could be used as an adjuvant for cancer immunotherapy. In mice modeling melanoma, administering fimH alongside a tumor antigen increased antigen-specific T...

Static anchor protein SNPH identified as target in spinal cord injury

DISEASE CATEGORY: Neurology INDICATION: Spinal cord injury (SCI) Inhibiting SNPH, a static anchor protein that holds axonal mitochondria stationary, could facilitate axonal regeneration to treat SCI. In mice with cervical (C5) dorsal hemisection-induced SCI, systemic...

Tau identified as target for autism associated with epilepsy syndromes

DISEASE CATEGORY: Neurology INDICATION: Autism Reducing levels of tau could reduce autistic behaviors associated with Dravet syndrome and cortical dysplasia-focal epilepsy syndrome. The syndromes are characterized by severe early-onset seizures, premature mortality, cognitive abnormalities and...

Preventing HPV infection with a capsid protein-based peptide

DISEASE CATEGORY: Infectious disease INDICATION: Human papillomavirus (HPV) A cell-penetrating peptide containing a sequence from the HPV L2, a capsid protein involved in intracellular viral trafficking, could prevent HPV infection. The HPV L2 sequence in...

IRE1 activation to treat cystic fibrosis and Pendred syndrome

DISEASE CATEGORY: Pulmonary; otologic INDICATION: Cystic fibrosis (CF); hearing loss Activating IRE1 could treat diseases caused by defective trafficking of membrane proteins to the cell surface, including CF and Pendred syndrome. Pendred syndrome is an...

Growth-accommodating biomimetic prosthetic valves for congenital valve disease

DISEASE CATEGORY: Cardiovascular INDICATION: Cardiovascular A biomimetic prosthetic valve designed to adapt to developmental growth could improve post-valve replacement outcomes in patients with congenital heart valve disease. Available prosthetic valves have a fixed diameter and...

CDC42BPA inhibition for high-grade serous ovarian carcinoma

DISEASE CATEGORY: Cancer INDICATION: Ovarian cancer Inhibition of CDC42BPA alone or in combination with carboplatin and PAK1 inhibition could treat high-grade serous ovarian carcinoma (HGSOC). Among 594 patient tumors, 44% of the samples had genomic...

Hyaluronic acid, collagen to prevent intervertebral disc degeneration after herniation

DISEASE CATEGORY: Musculoskeletal INDICATION: Musculoskeletal An injectable therapy comprising hyaluronic acid and a high-density collagen patch could prevent acute intervertebral disc degeneration after herniation as well as subsequent discectomy. The strategy involves injecting a hexadecyl...

Blocking STING to lower sepsis mortality

DISEASE CATEGORY: Infectious disease; cardiovascular INDICATION: Sepsis; block coagulation Targeting STING, a facilitator of innate immune signaling, could reduce bacterial sepsis mortality associated with disseminated intravascular coagulation (DIC). Among 40 patients with bacterial sepsis, high...

Targeting the NCK1-WIPF1 interaction for enteropathogenic E. coli infection

DISEASE CATEGORY: Infectious disease INDICATION: Escherichia A peptide that blocks the interaction between NCK1 and WIPF1 could treat or protect against enteropathogenic E. coli infection. A key step of infection involves binding of the SH3.2...

Targeting curli amyloid fiber subunits csgA and csgB to treat Salmonella

DISEASE CATEGORY: Infectious disease INDICATION: Salmonella A mAb targeting a pan-amyloid epitope on Salmonella enterica csgA and csgB could treat infections by the bacteria by disrupting biofilm formation. In cultured, pre-established S. enterica biofilms, the...