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The new editing techniques of Gene


A published in the international journal Science research paper, researchers from the American Southwest Medical Center, through research, the use of a novel gene editing technology successfully blocked Duchenne muscular dystrophy in the young mice (DMD) of progress. If the technology can be effectively and safely used in DMD patients, then it or to help develop the first gene-based therapies to treat editing human fatal disease


DMD is a serious boy in a common form of muscular dystrophy, the main features of progressive muscle degradation and exhibit weakness, the occurrence of the disease is mainly associated with the X chromosome DMD gene mutation caused the gene can encoding dystrophin, according to CDC data show that in 3500-5000 there is one boy who is in DMD patients, and the disease eventually will lead to individual patients had died before the age of 30.


In this paper, the researchers successfully used a gene-editing techniques to permanently correct mutations caused by DMD young mice, the researchers Eric Olson said the gene editing technology differs from other therapeutic means, because it can lead to the complete elimination DMD etiology. Early in 2014 we will make use of CRISPR / Cas9 mediated gene editing technology successfully corrected the mutation in the germ cells of mice to successfully suppress the occurrence of muscular dystrophy, which for the development of new therapies based on novel genome editing to DMD treatment foreshadowed, it also brings a great challenge for the gene editing technology used in the clinic.


In humans, germ cell editing is not feasible, but we need to develop the transport gene editing component to the policy body tissues after birth; researchers then by adeno-associated virus 9 (AAV9) to be transported to a small gene editing component mice, using the technique in mice treated DMD will produce dystrophin, and the function and structure of the body's skeletal muscles and heart in mice also occurs constantly improving. Leonela Amoasii said, AAV9 will be in a tissue-specific manner to effectively infect the human organism, but it does not cause human illness, which is just a molecule gene therapy as a missile.


In this paper, the researchers in DMD patients and a large number of preclinical animal models confirmed the effect of the new gene editing technology, so that researchers can use the genome editing technology to correct DMD caused by genetic mutations, which will accelerate human therapeutics such as DMD and other fatal disease process; the researchers hope that the results obtained can quickly late in the DMD mouse model of DMD patients quickly transformed into a human organism.