Salk Institute researchers have developed a new tool -- dubbed SATI -- to edit the mouse genome, enabling the team to target a broad range of mutations and cell types. The new genome-editing technology could be expanded for use in a broad range of gene mutation conditions such as Huntington's disease and the rare premature aging syndrome, progeria.
Research led by Dr. Krystof Bankiewicz, who recently joined The Ohio State University College of Medicine, shows that gene replacement therapy for Niemann-Pick type A disease is safe for use in nonhuman primates and has therapeutic effects in mice. These findings will publish online in the journal Science Translational Medicine. Prior to joining Ohio State as a professor of neurosurgery, Bankiewicz conducted this translational gene therapy research at the University of California at San Francisco.
Dr. Mihue Jang's group at the Korea Institute of Science and Technology(KIST) announced that they have developed a new gene editing system that could be used for anticancer immunotherapy through the simultaneous suppression of proteins that interfere with the immune system expressed on the surface of lymphoma cells and activation of cytotoxic T lymphocyte, based on the results of joint research conducted with Prof. Seokmann Hong's group at Sejong University
'The role of mitochondria has been largely ignored in the field of regenerative medicine, but earlier efforts in our lab suggested that they may affect the outcome of stem cell transplants,' said Tobias Deuse, M.D., the Julien I.E. Hoffman Chair in Cardiac Surgery at UCSF and lead author of the new study, published Aug. 19 in Nature Biotechnology.
In a paper published August 15 in the journal Neuron, the researchers describe a technique that uses a special version of CRISPR developed at UCSF to systematically alter the activity of genes in human neurons generated from stem cells, the first successful merger of stem cell-derived cell types and CRISPR screening technologies.
An optimized and newly engineered form of the adeno-associated vector 9 (AAV9) vec-tor used to deliver the galactosylceramidase gene to a mouse model of the inherited neu-rogenerative and rapidly fatal form of Krabbe disease improved clinical symptoms and prolonged median survival by 275%.
The All of Us Research Program at the National Institutes of Health has made strong progress in its efforts to advance precision medicine, according to a paper in the New England Journal of Medicine. With information provided by volunteers across the United States, All of Us is developing a robust data platform to support a wide range of health studies. The program aims to include data from 1 million or more people from diverse communities.
Scientists at Trinity College Dublin today announced a major breakthrough with important implications for sufferers of a common eye disease -- dry age-related macular degeneration (AMD) -- which can cause total blindness in sufferers, and for which there are currently no approved therapies.
City of Hope researchers have identified a potential combination targeted therapy for a deadly type of leukemia found in some infants, a population too young to receive full-blown chemotherapy. Called 'mixed lineage leukemia (MLL)-rearranged B cell acute lymphoblastic leukemia (B-ALL),' this blood cancer subtype comes with bleak health outcomes (overall survival rate is less than 50%), disease recurrence and development of resistance to existing therapies.
While watching the production of porous membranes used for DNA sorting and sequencing, University of Illinois researchers wondered how tiny steplike defects formed during fabrication could be used to improve molecule transport. They found that the defects -- formed by overlapping layers of membrane -- make a big difference in how molecules move along a membrane surface. Instead of trying to fix these flaws, the team set out to use them to help direct molecules into the membrane pores.