Acute myeloid leukemia (AML) harboring KMT2A rearrangements (KMT2A-r) represents a highly aggressive disease subtype, characterized by poor therapeutic response and a high risk of relapse.
DNA damage repair enzymes are interesting targets in cancer, since genomic instability and DNA repair defects are important cancer cell hallmarks. Poly (ADP-ribose) glycohydrolase (PARG) is the dominant eliminator of PARylation in the cell, the activity of which prevents excessive accumulation of PARylation, and promotes the dissociation of repair proteins, as well as ensuring the smooth completion of DNA repair process.
Oncolytic viral therapy offers new avenues in anticancer treatment, and two herpes simplex virus (HSV)-1-based virotherapies have recently gained approval for the treatment of advanced melanoma and recurrent glioblastoma.
Werner syndrome ATP-dependent helicase (WRN) is an enzyme involved in DNA replication and repair and has been identified as a synthetic lethality target in tumors with high microsatellite instability (MSI-H).
Quralis Corp. has entered into a number of agreements with the aim of advancing the treatment of fragile X syndrome, a genetic condition caused by a mutation of a single gene – fragile X messenger ribonucleoprotein 1 (FMR1) – on the X chromosome.
Microsatellite instability (MSI), which results from defects in the DNA mismatch repair system, is an important biomarker in colorectal cancer. While the MSI-high (MSI-H) status predicts response to pembrolizumab, a commonly used immune checkpoint inhibitor in metastatic colorectal cancer, a subset of MSI-H patients still does not respond to this treatment.
Gene and cell therapies (GCTs) can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them.
Suzhou Zion Pharma Technology Co. Ltd. has described transcription activator BRG1 (SMARCA4; BAF190A; SNF2-β) and/or probable global transcription activator SNF2L2 (SMARCA2; BAF190B; SNF2-α) inhibitors reported to be useful for the treatment of cancer and viral infections.
RSS
