Posted by on February 6, 2018 7:09 am
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Source: Sci Adv:blockbuster! Scientists use CRISPR/Cas9 technology for successful treatment of Duchenne muscular dystrophy

features session: 2018 gene editing and gene therapy seminar

2018 2 June 5 hearing /bio ValleyBIOON/ –recently, a study published in the International JournalScience Advanceson the study, from the United States and German scientists through the research describes a new CRISPR method, this new method may be expected to use of Duchenne muscular dystrophy(DMD) Disease patients the body more capable ofstem cellsto produce healthy myocardium, this new method also overcomes the earlier researchers of the DMD cells for gene editing encountered a variety of problems.

Photo credit: UT Southwestern Medical Center

DMD is a progressive disease, which can continue to“plunder”the patient’s muscle tissue, engaged in to induce the patient at a young age of heart failure and death, when the X-linked dystrophin gene is a large number of mutations, replication, and deletion will be induced by the DMD, and the X-linked dystrophin gene is mainly responsible for the produce to maintain muscle health and strong muscular dystrophy protein dystrophin protein in.

since the disease is based on mutations induced, and therefore it may be able to become the CRISPR technology is successful treatment of the next plague, but the problem is the disease is often based on a large number of mutations nearly 3000 of the produce, in order to overcome this problem, researchers use a method called“myoediting”technique, in this technique, the CRISPR mainly will be targeting the role of mutations in hot spot region, which can be effective for the entire gene clusters for editing and repair.

In this study, the researchers also from DMD patients the body the body has developed induced pluripotentstem cells, then these cells are carried out specific repair and reprogramming so that it can grow into cardiac cells, and finally produce dystrophin; and by providing a plurality of cells to build a“scaffold”that the researchers will be able to produce a small amount of myocardial tissue, at the same time they also found that the generation of myocardial tissue not only capable of beating, also be due to the muscular dystrophy protein to produce and continuously maintain health.

this study suggests that researchers develop new technologies or be able to treat up to 60%of DMD patients, and now the technology is in vivo in mice and dogs in vivo test, subsequent researchers found that mice without the diseased state has survived a year, the current researchers are using human cells to test them for future research the results expressed very optimistic, andclinical trialsperhaps in the future a few years to start. (生物谷Bioon.com

origin:

Chengzu Long, Hui Li, Malte Tiburcy, et al. Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing. the Science Advances  31 Jan 2018: Vol. 4, no. 1, eaap9004 DOI: 10.1126/sciadv. aap9004

Published at Fri, 06 Feb 2018 06:52:51 +0000

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