Shwachman-Diamond syndrome is an inherited bone marrow failure syndrome characterized by exocrine pancreatic insufficiency, cytopenia, growth restriction and skeletal abnormalities, and for which primary treatment is allogeneic hematopoietic stem cell transplantation, which is associated with significant toxicity.
The brain is a vulnerable organ that is compromised in the absence of energy production. Reduced fuel delivery to the brain correlates with aging and is an early predictor of potential neurological dysfunction, such as what occurs in Parkinson’s disease.
The mechanisms contributing to prostate cancer progression are still incompletely understood. There has been increasing attention on myosin heavy chain 6 (MYH6) for its involvement in cellular processes beyond the heart.
Antimicrobial peptides, existing in animals and plants, are the first line of defense of the organisms against bacteria. Thymol and carvacrol are two monoterpenoids with known antibacterial activity exerted through bacterial cell membrane structural disruption, which contributes to a lower risk of developing resistance compared to antibiotics acting against specific targets on cells.
Hematopoietic progenitor kinase 1 (HPK1) plays a relevant role in regulating T-cell, B-cell and dendritic cell function. As its inactivation both in vitro and in vivo contributes to tumor growth inhibition, HPK1 is considered a therapeutic target to explore in cancer immunotherapy.
Trueline Therapeutics Inc. and Broad Institute Inc. have reported preclinical data for the novel myeloid cell leukemia 1 (MCL1) inhibitor, BRD-810, being developed for the treatment of cancer.
Itheos Therapeutics Inc. recently presented the discovery of a first-in-class, potent and selective ENT1 inhibitor, EOS-984 (EOS-301984), for the treatment of solid tumors. Immune checkpoint inhibitors (ICIs) have become essential in treating solid tumors, but only a limited number of patients currently benefit from approved ICIs.
Aging is part of the life cycle and, although the effects are not manifest until after adulthood, it actually occurs from birth. The concept of senescence has traditionally been associated with aging. However, an embryo has senescent cells. In that case, what is aging, how can it be measured, and from what point in the life cycle?
Korean Research Institute of Bioscience and Biotechnology and Korea Research Institute of Chemical Technology have jointly developed molecular glue degraders acting as zinc finger protein Helios (IKZF2) degradation inducers. They are described as potentially useful for the treatment of cancer.
An Uppthera Inc. patent describes new proteolysis targeting chimera (PROTAC) compounds comprising E3 ubiquitin ligase-binding moiety covalently linked to a serine/threonine-protein kinase PLK1 (STPK13)-targeting moiety; they are reported to be useful for the treatment of cancer and neurological disorders.
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