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A pediatric immunologist at The Children's Hospital of Philadelphia collaborated with European gene therapy researchers who achieved marked clinical improvements in two young children with Wiskott-Aldrich syndrome, a very rare but often severe i...
Researchers have developed a new system to improve the delivery of genes, which could have the potential cure for several genetically transmitted diseases. Under the direction of Prashant Kumta, a professor of materials science, engineering and biomedical engineering, researchers are creating nano-particles capable of delivering DNA-based therapies for potential use in a variety of cancers and several genetic diseases. "We have developed a new system that will help physicians deliver their genetic life-saving payloads into enough cells to do some good," said Kumta, who has applied for a patent on the non-viral gene delivery system.
A molecule that naturally degrades a protein linked to Alzheimer's disease appears to reduce the levels of that protein by nearly 50 percent when delivered by gene therapy, researchers at the Salk Institute and UC San Diego have found in collaboration with researchers at the University of Kentucky. The findings appear in the March 15 issue of the Journal of Neuroscience.