In a paper titled "BBB opening with focused ultrasound in nonhuman primates and Parkinson's disease patients: Targeted AAV vector delivery and PET imaging," published in Science Advances, researchers led by the University ...
Metachromatic leukodystrophy is a rare genetic disorder that mainly affects young children and results in severe neurological symptoms accompanied by a loss of motor and intellectual capacities. At Paris Brain Institute, ...
Apr 22, 2024
0
8
Scientists at City of Hope, one of the largest cancer research and treatment organizations in the United States, show that a COVID-19 vaccine developed at the organization's Los Angeles campus also protects against mpox, ...
Mar 4, 2024
0
39
Researchers have revealed the mechanism of a drug shown to be effective in treating certain types of cancer, which targets a protein modification silencing the expression of multiple tumor suppressor genes. They also demonstrated ...
Feb 21, 2024
0
20
Indiana University School of Medicine researchers have uncovered vital insights regarding a liver trigger that blocks an undesired immune response from gene therapy, surprisingly resulting in activating specific immune cells ...
Dec 7, 2023
0
29
Doug Millay, Ph.D., a scientist with the Division of Molecular Cardiovascular Biology at Cincinnati Children's has dedicated his career to revealing the most fundamental mechanisms of skeletal muscle development. He has been ...
Apr 28, 2023
0
79
Research led by Randal J. Kaufman, Ph.D., has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases. ...
Dec 7, 2022
0
47
In a new paper, University of California, Irvine researchers explain how precision genome editing agents have enabled precise gene correction and disease rescue in inherited retinal diseases (IRDs). The study, titled, "Precision ...
Sep 23, 2022
0
60
In a major new development in the quest to develop better gene therapies with which to treat a host of diseases, researchers at the University of Massachusetts Amherst and UMass Chan Medical School recently announced that ...
Jul 25, 2022
0
42
Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye disease. Researchers from the National Eye Institute ...
Dec 9, 2021
0
60
