A recent patent from Korea University Research & Business Foundation Sejong Campus describes quinazoline-benzimidazole derivatives acting as microtubule destabilizers (tubulin polymerization inhibitors) and HER2 inhibitors reported to be useful for the treatment of cancer.
Branchio-Oto-Renal syndrome 1 (BOR1) is caused by pathogenic variants in the EYA1 gene, and the gene behind the pathogenesis of BOR2 is SIX5. Growing evidence exists regarding GATA and PAX-SIX-EYA-DACH transcriptional networks playing a key role in normal development. A case report of a patient harboring a new variant in the DACH1 gene was recently presented.
Polyglutamine (polyQ) diseases, a group of dominantly inherited CNS disorders, are caused by an abnormal expansion of cytosine-adenine-guanine repeats (usually over 35-40 repeats). PolyQ diseases, including spinocerebellar ataxia and Huntington’s disease, cause brain neurodegeneration, leading to progressive motor and often cognitive signs.
The liver is a key at maintaining glucose and lipid homeostasis, crucial processes for metabolic health. When these processes are disrupted, a series of metabolic disorders may occur, including type 2 diabetes and metabolic dysfunction-associated steatotic liver disease (MASLD).
Researchers from First Affiliated Hospital of Zhengzhou University published results from a study that aimed to investigate the mechanisms by which programmed cell death and mitochondrial function influence prognosis and immunotherapy response in renal cell carcinoma (RCC), and to identify novel therapeutic targets for this disease.
Researchers from Suzhou Genassist Therapeutics Co. Ltd. have developed a novel canine model of Duchenne muscular dystrophy (DMD), expected to be more predictive of disease pathogenesis and treatment efficacy.
Everest Medicines Ltd. has obtained IND clearance from the FDA for EVM-14, an off-the-shelf mRNA cancer vaccine targeting multiple tumor-associated antigens and designed to treat various cancers, including non-small-cell lung cancer and head and neck cancer.
Dengue virus (DENV) is a member of the flavivirus family causing dengue fever, a mosquito-borne disease that can be life-threatening. Despite DENV’s widespread presence in over 80 countries and the significant health burden posed by the infection, there is still a critical need for therapeutics and vaccines, with current treatment options only providing supportive care.
Entrada Therapeutics Inc. has received authorization from the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) and research ethics committee to initiate a phase I/II study of ENTR-601-45 for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the DMD gene amenable to exon 45 skipping.
Esophageal squamous cell carcinoma (ESCC) accounts for about 90% of esophageal cancer cases and is the sixth most common cause of cancer death worldwide. Targeting cancer stem cells offers a therapeutic approach for addressing cancer, concretely in those cases that are resistant to cisplatin.
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