New drug targets for Alzheimer’s identified from cerebrospinal fluid

Researchers at Washington University School of Medicine in St. Louis have linked disease-related proteins and genes to identify specific cellular pathways responsible for Alzheimer’s genesis and progression. The proteins were gathered from cerebrospinal fluid and are a good proxy for activity in the brain. Several of them may be potential targets for therapies.

Penn Medicine at the American Society of Gene and Cell Therapy 27th Annual Meeting

Researchers from the Gene Therapy Program (GTP) at the Perelman School of Medicine at the University of Pennsylvania will present ten research abstracts, two invited talks, and a workshop presentation highlighting their translational science and discovery research on gene therapy, gene editing, and adeno-associated virus (AAV) vector technology at the American Society of Cell and Gene Therapy (ASGCT) 27th Annual Meeting on May 7 – 11, 2024 in Baltimore, Maryland.

Brain tumor treatment by targeting TUG1, a gene that controls replication stress

A new study has unravelled a crucial link between how cancer cells cope with replication stress and the role of Taurine Upregulated Gene 1 (TUG1). By targeting TUG1 with a drug, the researchers were able to control brain tumor growth in mice, suggesting a potential strategy to combat aggressive brain tumors such as glioblastomas.

Improved Gene Editing Method Could Power the Next Generation of Cell and Gene Therapies

A new approach to the genetic engineering of cells promises significant improvements in speed, efficiency, and reduction in cellular toxicity compared to current methods. The approach could also power the development of advanced cell therapies for cancers and other diseases, according to a study from researchers in the Perelman School of Medicine at the University of Pennsylvania.

Chula Researches “Tooth Loss” in Thai People- A Hereditary Condition Caused by Genetic Abnormalities

Chulalongkorn dental research reveals that Thais suffer more tooth loss than their foreign counterparts with 9 out of 100 Thais suffering the condition. One of the most important factors is hereditary gene abnormalities.

WVU researcher says gene discovery may lead to new tests, treatments for Alzheimer’s in women

Women make up two-thirds of Americans who have Alzheimer’s disease, yet scientists have yet to determine what makes them so susceptible to the condition. Bernard Schreurs, a researcher with the West Virginia University School of Medicine and Rockefeller Neuroscience Institute, directs the West Virginia Alzheimer’s…

Regulator Proteins or Symphonies of Genes: Statistical Modeling Points Way Toward Unified Theory for DNA Folding

Researchers seek to point a way toward a unified theory for how DNA changes shape when expressing genes. Presenting their work in Biophysics Reviews, the scientists use an approach called statistical mechanics to explore the phenomenon of so-called expression waves of gene regulation.

Darwin’s short-beak enigma solved

University of Utah biologists discovered that a mutation in the ROR2 gene is linked to beak size reduction in numerous breeds of domestic pigeons. Surprisingly, different mutations in ROR2 also underlie a human disorder called Robinow syndrome. The ROR2 signaling pathway plays an important role in the craniofacial development of all vertebrates.

New Insights Into Why People With Down Syndrome Are At Higher Risk For Leukemia

Scientists from Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago were the first to examine endothelial cells – one of the main sources of blood production – for clues as to why people with Down syndrome have higher prevalence of leukemia. They identified a new set of genes that are overexpressed in endothelial cells of patients with Down syndrome. This creates an environment conducive to leukemia, which is characterized by uncontrolled development and growth of blood cells. Their findings, published in the journal Oncotarget, point to new potential targets for treatment and possibly prevention of leukemia, in people with Down syndrome and in the general population.

New High-Throughput Method to Study Gene Splicing at an Unprecedented Scale Reveals New Details About the Process

Genes are like instructions, but with options for building more than one thing. Daniel Larson, senior investigator at the National Cancer Institute, studies this gene “splicing” process, which happens in normal cells and goes awry in blood cancers like leukemia.

Opening Up DNA to Delete Disease

Protein editorial assistants are clearing the way for cut-and-paste DNA editors, like CRISPR, to access previously inaccessible genes of interest. Opening up these areas of the genetic code is critical to improving CRISPR efficiency and moving toward futuristic, genetic-based assaults on disease. The DNA-binding editorial assistants were devised by a U.S.-based team of bioengineers, who describe their design in APL Bioengineering.