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 ...
Researchers from MIT, the McGovern Institute for Brain Research at MIT, the Howard Hughes Medical Institute, and the Broad Institute of MIT and Harvard have developed a new way to deliver molecular therapies to cells. The ...
Aug 19, 2021
0
440
When the Human Genome Project was completed, in 2003, it opened the door to a radical new idea of health—that of personalized medicine, in which disease risk and appropriate treatment would be gleaned from one's genetic ...
Mar 25, 2019
1
689
For decades, scientists have dreamed of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of fixing faulty genes with healthy ones, has held ...
Aug 29, 2024
0
43
Gene therapy could potentially cure genetic diseases, but it remains a challenge to package and deliver new genes to specific cells safely and effectively. Existing methods of engineering one of the most commonly used gene-delivery ...
Aug 8, 2024
0
18
A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those ...
Jul 17, 2024
0
52
Drug development is typically slow: the pipeline from basic research discoveries that provide the basis for a new drug to clinical trials to production of a widely available medicine can take decades. But decades can feel ...
Jun 27, 2024
1
19
A viral gene therapy developed by University at Buffalo researchers has reversed some brain abnormalities in infant mice with FOXG1 syndrome, a significant step toward one day treating children with this severe neurodevelopmental ...
Jun 13, 2024
0
23
In an important step toward more effective gene therapies for brain diseases, researchers from the Broad Institute of MIT and Harvard have engineered a gene-delivery vehicle that uses a human protein to efficiently cross ...
May 16, 2024
0
25
In a worldwide first-of-its-kind study published in the journal Nature Communications this week, a team of scientists from Children's Medical Research Institute (CMRI) have tested novel gene therapies in a whole human liver, ...
Mar 14, 2024
0
13
