"Sleeping Beauty" transposon is widely used in the transformation of the genome, and it shows great potential in clinical trials leukemia and lymphoma. EMBL scientists have recently solved the key structure of "Sleeping Beauty" transposon, and they greatly enhanced the efficiency of this transposon inserting genes by making improvements based on the structure. The study was published in the journal Nature Communications on March 31th.
In the treatment of leukemia and lymphoma, "Sleeping Beauty" transposon is used to insert genes to a patient's T cells. After transformation of these T cells back to the patient later, the ability to search and destroy cancer cells. This method is simpler than rely on viral vector gene therapy is much more, the cost is relatively low. For this reason, "Sleeping Beauty" transposon attracted many pharmaceutical companies, will soon enter clinical trials.
Therapeutic T cell transformation and selection takes time, which is a major bottleneck in gene therapy. T cell transformation and migration faster, better patient prognosis, treatment costs are lower. However, to date only people on the "Sleeping Beauty" transposon is modified based on speculation. To this end, EMBL researchers determined the structure of "Sleeping Beauty" active region.
Inserting gene therapy includes "Sleeping Beauty" transposon and CRISPR / Cas9 advantage. CRISPR / Cas9 cleave specific sites in the genome, is the ideal way to remove the genetic error. However, CRISPR / Cas9 cannot be directly inserted gene fragment, relying instead on cell repair mechanisms. "Sleeping Beauty" transposon can simply bring own fragment inserted into the genome, and its efficiency has been improved significantly. "We designed a variant of 30% higher efficiency than the existing version," who led the study Orsolya Barabas said.
Scientists have found that in May last year, were high hopes that gene therapy may not completely solve the problem as people expected. Two clinical trials leading medical journal "New England Journal of Medicine," published show that gene therapy to restore vision will fail within a few years.
In July last year, researchers at Harvard Medical School and Boston Children's Hospital by gene therapy, the success of genetic deafness in mice restored the hearing. The results are published in the journal Science Translational Medicine for the treatment of hearing loss due to mutations basis.
RNAi makes the cells degrade corresponding mRNA by introducing siRNA or shRNA and conducts genetic screening according to loss of function phenotype. However, RNAi is relatively prone to error. UCSF research team largely overcame the off-target effects of RNAi for genome-wide screening through the establishment of a complex mix of ultra-shRNA library. They further enhances the activity of the shRNA by improving the system to showcase a new generation of shRNA libraries which is targeted the human and mouse genomes. The results were published in the journal PNAS. Studies have shown that a new generation of RNAi library's performance in the test is equally compared with CRISPRi.
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