Traditionally, geneticists divide disorders into “simple,” where a single gene mutation causes disease, or complex, where mutations in many genes contribute modest amounts. A new study suggests that the truth is somewhere in between.
Addressing concerns about gene drive releases in the wild, UC San Diego scientists and their colleagues have developed two new genetic systems that halt or eliminate gene drives after release. The details are published in the journal Molecular Cell.
St. Jude researchers showed that an inherited variant of the GATA3 gene is tied to minimal residual disease levels and response to therapy for acute lymphoblastic leukemia.
With the aid of artificial intelligence, UC San Diego scientists have solved a long-standing puzzle in human gene activation. The discovery described in the journal Nature could be used to control gene activation in biotechnology and biomedical applications.
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.
Researchers at Ludwig Institute for Cancer Research and University of California San Diego School of Medicine report that inhibiting a key enzyme caused human cancer cells associated with two major types of breast and ovarian cancer to die and in mouse studies reduced tumor growth.
Genomic research helps explain why some people with a common TP53 mutation widespread in Brazil get cancer while others do not.
New NEI-supported research provides insight into the eye conditions associated with Marfan syndrome, where weakened zonule fibers cause vision problems.
St. Jude Children’s Research Hospital software enables detection of previously unknown cancer-causing gene fusions, pointing the way to new treatments.
New research highlights the RCAN1 gene’s effect on memory and learning.
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.
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.