About 10% of amyotrophic lateral sclerosis (ALS) cases result from inherited genetic mutations, with about 20% of them attributed to mutations in the gene encoding the ubiquitous cytoplasmic copper/zinc superoxide dismutase 1 (SOD1).
Lysine demethylase 4C (KDM4C) is a chromatin-modifying protein frequently overexpressed across multiple solid and hematological cancers (including breast, lung, colon, prostate, esophageal cancers and lymphomas) and has been linked to chromatin instability and enhanced cell proliferation and stem cell-like behavior.
Ovarian cancer is a highly lethal gynecological malignancy with limited therapeutic options for recurrent and drug-resistant disease. Sirtuin 2 (SIRT2) promotes tumor cell proliferation, migration and invasion by regulating multiple oncogenic signaling pathways. Although SIRT2 has emerged as a promising therapeutic target, conventional inhibitors often result in incomplete and transient suppression of its activity.
Opioid use disorder (OUD) causes high morbidity and mortality rates, with fentanyl driving unprecedented overdose rates. Researchers from the University of California have used an AI-based drug discovery platform with the aim of identifying preclinical drug candidates for OUD.
Hepatoblastoma is the most common liver cancer during childhood, with limited therapeutic options in aggressive or relapsed cases. NEDDylation is a post-translational modification that modulates cullin-RING ligases and has arisen as a crucial regulator of protein turnover in cancer.
Insilico Medicine Cayman Topco has obtained IND approval from the FDA for ISM-8969 for the treatment of Parkinson’s disease, enabling initiation of a phase I trial in healthy volunteers.
Researchers from Leiden University Medical Center previously demonstrated that the junction adhesion molecule-independent mutant (jin-3) reovirus, which is a double-stranded RNA virus not associated with severe human disease, presents extended tumor tropism compared to wild-type reoviruses.
The prevalence of asthma differs between men and women, and furthermore, the difference changes over the lifespan. “Asthma is more common in boys than girls, but more common in women than men,” Clare Lloyd told BioWorld. Females are particularly susceptible to asthma during developmental periods of hormonal changes, also known as puberty, pregnancy and menopause.
Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant skeletal muscle disorder with a prevalence of approximately 1 in 8,000. The disorder is driven by aberrant expression of double homeodomain protein 4 (DUX4) within the D4Z4 macrosatellite array. Currently, effective treatments for FSHD are lacking. Strategies aimed at reducing DUX4 expression could hold promise as potential therapeutic approaches for FSHD.
Recent evidence has suggested threonine tyrosine kinase (TTK) as a crucial element of the mitotic checkpoint for the correct functioning of spindle assembly checkpoint (SAC), making it a potential therapeutic target in cancer.
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