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Dravet syndrome is a rare and life-changing form of epilepsy. Dravet syndrome affects approximately 1 in 15,700 children, and most cases are caused by mutations in the SCN1A gene. This gene plays a critical role in the brain's ability to regulate activity through flash interneurons. The disease has long made scientists eager to develop more effective treatments due to severe seizures and developmental delays.
Immune checkpoint inhibitor (ICI) therapy has demonstrated therapeutic benefits and prolonged survival in cancer patients. However, most patients either fail to respond to ICI therapy or develop resistance to it.
In a new study, researchers from the Jackson Laboratory and UConn Health not only show how cancer hijacks this tightly regulated RNA splicing and rearrangement, but also propose a potential therapeutic strategy to slow down or even shrink aggressive and hard-to-treat tumors. The discovery could change the way people treat aggressive cancers, such as triple-negative breast cancer and certain brain tumors, for which current treatment options are limited.
Stem cells are immature cells that have a fundamental regenerative role in almost all tissues. They are usually in a quiescent, slowly dividing state. But after injury, they can repair damaged tissues by switching to an activated state so that they can rapidly proliferate and become mature, functional cells. For example, hematopoietic stem cells mostly reside in the bone marrow and remain quiescent until they are stimulated or "mobilized" into the blood.
Chimeric antigen receptor (CAR) T cell therapy is a promising cancer treatment, but how to enhance its efficacy has always been a mystery. Recently, in a research report titled "Cullin-5 deficiency promotes chimeric antigen receptor T cell effector functions potentially via the modulation of JAK/STAT signaling pathway" published in the international journal Nature Communications, researchers from Nagoya University and other institutions in Japan have discovered a way to improve the effectiveness of this potential cancer therapy. By modifying a specific gene, the ability of immune cells to fight cancer can be enhanced for a long time, which may reduce the chance of cancer recurrence.
In the field of cancer treatment, proteolysis targeting chimeras (PROTACs) are gradually emerging as a new generation of drugs. This type of drug can accurately target and degrade proteins closely related to cancer growth, bringing hope for conquering those "undruggable" targets that are difficult to deal with with traditional drugs, and also opening up new treatment pathways for many diseases that have no effective treatment options. However, the intracellular transport mechanism of PROTACs, especially what factors affect its therapeutic effect in cancer cells, has always been a mystery that researchers are eager to solve.
In the field of medical research, ACE2 has attracted much attention as a receptor for SARS-CoV-2. It is also of great significance during pregnancy. The circulating level of ACE2 in pregnant women is higher than that in non-pregnant women, and the expression and genetic variation of ACE2 are closely related to various pregnancy diseases such as preeclampsia and fetal growth restriction. Recently, a research article titled "Genetically edited human placental organoids cast new light on the role of ACE2" published in Cell Death Dis constructed a placental organoid model through gene editing technology, and deeply explored the mechanism of action of ACE2 in placental development.
In 2010, Sonia Vallabh witnessed her 52-year-old mother develop a rapidly progressive, mysterious, and undiagnosed dementia, and soon died from it. A year later, she learned that her mother had a hereditary prion disease, fatal familial insomnia. After undergoing genetic testing, Sonia learned that she also carried the disease-causing gene mutation, which meant that she herself was likely to suffer from this prion disease. More importantly, this fatal disease usually develops around the age of 50 and quickly leads to death, and there is no cure.
Dysfunction of DNA repair is a key driver of cancer. Understanding the molecular mechanisms behind dysfunctional DNA repair in cancer cells is crucial for the occurrence of cancer and the development of new therapies. Recently, in a research report titled "EZH2 directly methylates PARP1 and regulates its activity in cancer" published in the international journal Science Advances, scientists from Northwestern University and other institutions discovered a new molecular mechanism behind dysfunctional DNA repair in prostate cancer cells through research. This research finding is expected to guide scientists to develop new targeted therapies to treat prostate cancer patients who are resistant to current standard therapies.
CRISPR-Cas9 has long been likened to a pair of genetic scissors because of its ability to elegantly and precisely snip any desired DNA fragment. But it turns out that the CRISPR system has more than just one strategy in its toolbox. CRISPR is a mechanism originally discovered in bacteria, and it has been operating for centuries as an adaptive immune system. Certain single-celled organisms naturally use CRISPR to protect themselves from viruses (called bacteriophages) and other foreign genetic fragments.
