Targeted exon skipping with AAV-mediated split adenine base editors
Abstract Techniques for exclusion of exons from mature transcripts have been applied as gene therapies for treating many different diseases. Since exon skipping has been
Abstract Techniques for exclusion of exons from mature transcripts have been applied as gene therapies for treating many different diseases. Since exon skipping has been
Amyotrophic lateral sclerosis (ALS) is a debilitating and fatal disorder that can be caused by mutations in the superoxide dismutase 1 (SOD1) gene. Although ALS
The Food and Drug Administration (FDA or Agency) plays a critical role in protecting the United States from public health threats including the Coronavirus Disease
Abstract Vascularized composite allotransplantation (VCA) encompasses face and limb transplantation, but as with organ transplantation, it requires lifelong regimens of immunosuppressive drugs to prevent rejection.
For years, scientists have predicted that 3-D printing—which has been used it to make toys, homes, scientific tools and even a plastic bunny that contained
A 34-year-old woman has given birth thanks to a new fertility procedure that involved collecting, maturing, and freezing some of her eggs five years earlier.
In a recent study of more than 900 rats, Shelly Flagel and her team of neuroscientists at the University of Michigan found that a region
The identification of artemisinin (ART) in 1971 allowed treatment of malaria resistant to chloroquine, the prevailing drug at the time, and provided hope for a
Teams at Helmholtz Zentrum München, LMU Munich and the Technical University of Munich (TUM) have developed a new algorithm that enables automated detection of metastases
Trophoblasts, cells present during development in the fetus and placenta that also circulate in a pregnant woman’s bloodstream, could potentially be used for noninvasive prenatal
About half a million people, most of them children living in Africa, are killed each year by malaria1. Management of malaria, particularly that caused by
Researchers have engineered Escherichia coli bacteria to make energy exclusively from carbon dioxide, according to a paper published today (November 27) in Cell. E. coli
Mosquitoes infected with Wolbachia bacteria and released into the wild are associated with a sharp decrease in dengue fever infections in humans, scientists reported Thursday
Variation in the DNA sequences that constitute the blueprint of life is essential to the fitness of any species, yet thousands of DNA alterations are
Preliminary research suggests that using CRISPR to treat cancer is safe in humans and could become a feasible therapeutic method in the future, although its
Altered energy metabolism is a hallmark of malignancy that can be harnessed to detect and treat cancer1. But tumours are metabolically diverse, so, for treatments
Brain organoids, small pieces of the human cerebral cortex grown in the lab, are becoming valuable scientific tools. By modeling the growth of brain cells
The mammalian brain consists of billions of neurons wired together in various circuits, each one involved in specific physiological functions. To better understand how these
The human brain has undergone substantial change since humans diverged from chimpanzees and the other great apes1,2. However, the genetic and developmental programs that underlie
One day, doctors may be able to use the metabolites in blood samples to predict the likelihood of a person surviving another five to 10
A mesh-like network of cells within mice’s skin plays a previously unknown role in pain perception, researchers reported today (August 16) in Science. The scientists
When Sara Campbell first typed “exercise and microbiome” into PubMed in 2010 and hit enter, “the search literally said zero,” she tells The Scientist. Campbell
Despite the recent approval of two cancer therapies that use CAR T cells to treat lymphoma, 25 percent of eligible patients still choose to enter
People with a rare form of inherited blindness are being enrolled for the world’s first in vivo study of a CRISPR-based therapy, the Associated Press
Senators Dianne Feinstein (D-CA), Marco Rubio (R-FL), and Jack Reed (D-RI) introduced a resolution yesterday (July 15) calling for global collaboration in developing guidelines for
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