Liu then modified the enzymes to make them more precise and fused them to CRISPR to create fusion proteins called "base editors." Since CRISPR technology is good at reading DNA and finding a target, it can effectively deliver the editors to the gene that needs to be changed. It's important...
Gene therapy holds promise for the treatment of these monogenic neuromuscular diseases, and many such therapies have made substantial strides toward clinical translation. Recently, genome engineering tools, including targeted gene editing and gene regulation, have become available to correct the underlying ...
The current types of gene therapy for hemophilia mainly utilize techniques to ectopically express a normal gene by a vector, while leaving the abnormal somatic gene as it is. The strategies of gene therapies for hemophilia are mainly categorized into two approaches: (i) direct injection of a vec...
The set of regulations on promoting the cell and gene therapy industry is the first such legislation in China. Under the legislation, patients with severe conditions are able to use new medicines undergoing clinical trials. The regulations encourage enterprises and research institutes to participate in...
CRISPR/Cas9 is a unique technology that enables geneticists and medical researchers to edit parts of the genome by removing, adding or altering sections of the DNA sequence. The study, published inTheranostics, demonstrates the potential of this innovative platform to revolutionize gene therapy and pr...
Gene therapy is a powerful developing technology that has the potential to address myriad diseases. For example, Huntington's disease, a neurodegenerative disorder, is caused by a mutation in a single gene, and if researchers could go into specific cells and correct that defect, theoretically those...
the application potential of T cell therapy has increased due to the development of CAR-T cell therapy. Similar to TCR-T cell therapy, CAR-T cell therapy uses gene transduction techniques (retrovirus, lentivirus, non-viral vector, etc.) to confer T cells the ability to precisely attack tumors...
"We are getting closer to solving the problem of cell delivery. This has been something that's limited medicine and gene therapy for a century," said Smith, who also is a professor in the Carle Illinois College of Medicine. "I think that the ability to track and analyze delivery incells...
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The therapy could be used alongside chemotherapy to treat early-stage breast cancer tumors before they spread, according to Natalie Artzi, a principal research scientist at MIT’s Institute for Medical Engineering and Science (IMES) and an assistant professor of medicine at Brigham and Women’s Hos...