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CDK7 inhibition for treating small cell lung cancer

DISEASE CATEGORY: Cancer INDICATION: Lung cancer Inhibiting CDK7, a cell cycle regulator, could treat small cell lung cancer (SCLC) and enhance the efficacy of checkpoint inhibition and chemotherapy. In mouse and human SCLC cell lines,...

Agonizing the checkpoint protein VISTA for systemic and cutaneous lupus

DISEASE CATEGORY: Autoimmune disease INDICATION: Lupus VISTA agonism could treat systemic lupus erythematosus (SLE) and discoid lupus erythematosus (DLE), the most common type of cutaneous lupus. Levels of the inhibitory checkpoint protein VISTA in skin...

SNCA mRNA-binding compound for Parkinson's

DISEASE CATEGORY: Neurology INDICATION: Parkinson's disease (PD) Matthew Disney and his colleages have generated a small molecule targeting SNCA mRNA, preventing α synuclein protein production, that could treat PD. Structure-guided design for binding to the...

TNFR2 and IL-33 identified as targets in Dupuytren's disease

DISEASE CATEGORY: Musculoskeletal INDICATION: Dupuytren's disease Inhibiting TNFR2 and IL-33 could treat Dupuytren's disease, which is characterized by progressive fibrosis of the palmar and digital fascia. The effector cells in Dupuytren's, the myofibroblasts, mediate the...

Supplementation with for alcoholic fatty liver disease

DISEASE CATEGORY: Hepatic INDICATION: Liver disease Oral administration of the gut microbe Roseburia intestinalis could treat alcoholic fatty liver disease. In 22 patients with alcoholic liver disease, lower abundances of Roseburia spp. in fecal samples...

CRISPR correction of CFTR mutations to generate autologous transplants to treat cystic fibrosis

DISEASE CATEGORY: Pulmonary INDICATION: Cystic fibrosis (CF) Autologous transplants for CF patients generated from patient-derived upper-airway basal stem cells edited via CRISPR-Cas9 could treat the genetic disease. The transplants were created by: culturing 3-D organoids...

Inhibiting SPK2 to treat Middle East respiratory syndrome

DISEASE CATEGORY: Infectious disease INDICATION: Coronavirus SKP2 inhibition could treat Middle East respiratory syndrome (MERS) coronavirus infection by reducing degradation of BECN, an autophagy regulator. In cell-based assays, MERS coronavirus infection induced SKP2 phosphorylation and...

Targeting SMPD1 to treat norovirus

DISEASE CATEGORY: Infectious disease INDICATION: Norovirus Inhibiting the sphingomyelinase SMPD1, by preventing its activation or blocking its release from lysosomes, could treat norovirus infection. Norovirus infect cells via uptake by endosomes, which undergo acidification --...

FERMT2 identified as target in renal fibrosis

DISEASE CATEGORY: Renal INDICATION: Renal damage Inhibiting the focal adhesion molecule FERMT2 could treat renal fibrosis. In renal tissue from patients with renal fibrosis, FERMT2 levels were higher in fibrotic tissue than in normal tissue....

Crry gene therapy to treat multiple sclerosis

DISEASE CATEGORY: Autoimmune disease INDICATION: Multiple sclerosis (MS) Adeno-associated viral vector (AAV)-mediated overexpression of the complement inhibitor Crry at C3-bound synapses could treat MS. The AAV9 vector encodes Crry fused to a domain of CR2,...

Promoting RNF208 expression in metastatic breast cancer

DISEASE CATEGORY: Cancer INDICATION: Breast cancer Increasing the expression of RNF208, an estrogen-inducible E3 ligase, could treat metastatic triple-negative breast cancer (TNBC). RNA levels of RNF208 were lower in tumor tissue from patients with TNBC...

CLEC14A-positive stem cell transplants for muscle regeneration

DISEASE CATEGORY: Musculoskeletal INDICATION: Musculoskeletal Transplantation with muscle-derived CLEC14A-positive stem cells could regenerate muscle following injury, despite lacking expression of known myogenic regulator PAX7. The myogenic cells were isolated from muscle fiber fragments that underwent...

Hydrogel-delivered, thrombin-derived peptide to treat and prevent Staphylococcus and Pseudomonas infections

DISEASE CATEGORY: Infectious disease INDICATION: Staphylococcus ; Pseudomonas  A polymer-based hydrogel containing the thrombin-derived peptide TCP-25 could treat and prevent S. aureus and P. aeruginosa infections. A hydrogel formulation of 0.1% (0.3nM) TCP-25, which was...

IL1RL1 in Treg cells as a lung cancer target

DISEASE CATEGORY: Cancer INDICATION: Lung cancer Inhibiting IL1RL1, which signals in response to IL-33 binding, in Treg cells could treat lung cancer. In single-cell RNA sequencing analyses of CD4 + T cells from the lymph...

Targeted Bcl-XL degrader identified to treat ALL, TNBC and SCLC

DISEASE CATEGORY: Cancer INDICATION: Acute lymphoblastic leukemia (ALL); breast cancer; small cell lung cancer A University of Florida team led by Dialectic Therapeutics Inc. co-founders Guangrong Zheng and Daohong Zhou showed the company’s lead candidate,...