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Investigators report no evidence of toxicity in the four hemophilia B patients enrolled to date in a gene therapy trial using a vector under development at St. Jude Children's Research Hospital and UCL (University College London) to correct the inhe...
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.