crispr/cas9 can cut the genome at a specific site,This is the ideal way to eliminate genetic errors.This method is much simpler than gene therapy that relies on viral vectors
In July last year, researchers at Harvard Medical School and Boston Children's Hospital passed gene therapy.Successfully restored the hearing of mice with hereditary deafness.The cost is also relatively low.
The University of California research team in San Francisco has built an ultra-complex hybrid shrna library,To a large extent, When used for genome-wide screening,It overcomes the off-target effect of rnai.and so,The "Sleeping Beauty" transposon has attracted many pharmaceutical companies,quickly,It entered clinical trials.The faster the transformation and transplantation of T cells,The prognosis of the patient is better,The cost of treatment is also lower.
research shows,The performance of the tested new generation of rnai library is comparable to crispri.The "Sleeping Beauty" transposon can insert the fragments it carries directly into the genome,And its efficiency has been significantly improved."The variant we designed is 30% more efficient than the current version.but,crispr/cas9 cannot directly insert gene fragments,It depends on the repair mechanism of the cell. "Sleeping Beauty" transposon is widely used for genome modification,It has shown great potential in clinical trials of leukemia and lymphoma.This result has been published in the pnas journal, Proceedings of the National Academy of Sciences.Two clinical trials published in the top medical journal New England Medicine show thatGene therapy aimed at restoring vision will fail in a few years.After these modified T cells return to the patient,Able to search and destroy cancer cells."Orsolya barabas, Who led this research, Say.(.They further enhanced shrna's activities through system improvements,Show people the new generation shrna library for human and mouse genomes.The results were published in the journal Science Translational Medicine,它为治疗由基因突变引起的听力损失奠定了基础。
在插入治疗基因方面,像“睡美人”这样的转座子具有优于crispr / cas9的优势。embl的科学家最近分析了“睡美人”转座子的关键结构,并在此基础上进行改进,转座子插入基因的效率大大提高。 在治疗白血病和淋巴瘤时, “睡美人”转座子用于将基因插入患者的t细胞
治疗性t细胞的转化和选择需要时间,这是基因治疗的主要瓶颈。 去年五月 科学家发现基因疗法希望它不能完全解决人们期望的问题