Nature sub-journals:the use of CRISPR/Cas9 to enhance gene therapy treatment of genetic disease potential

Source: Nature sub journal:the use of CRISPR/Cas9 to enhance gene therapy treatment of genetic disease potential

2018 years 4 months 19 days/bio ValleyBIOON/—gene therapy has the potential treatment ofgeneticdisease, however, make the scientists feel frustrated one of the main problems still exist: the use of a healthy gene to replace“bad”genes are often just briefly repair. In the normal case, the healthy alternative gene only in a few weeks to play a role.< p>now, in a new study, from Washington University in St. Louis School of Medicine researchers in the gene editing tool CRISPR/Cas9 with an inactivated virus combined with the healthy gene delivery into live mouse in vivo the precise location. More importantly, these researchers confirmed the delivery of the healthy gene in mice maintained properly active for at least 6 months. According to their argument, this type of gene expression, duration is usually four to six weeks. Them in the six months ended for the mice experiments, but they indicate that this gene expression duration equivalent to this repair in mice last a lifetime. Related research results recently published in the Gene Therapy journals, thesis entitled,“Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9”.
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pictures from CDC/G. William Gary, Jr.

Thesis communication the author, radiationtumorProfessor David T. Curiel, Dr. said,“over the years, gene therapy is the presence of a limit is difficult to achieve long-term gene expression to treat disease. We confirmed that long-term expression of α-1-antitrypsin encoding gene can be treated with alpha-1-antitrypsin deficiency（alpha-1-antitrypsin deficiency, Alpha-1-anti-trypsin deficiency is the most common hereditary emphysema form. Today, we are the application of this technology in the treatment of hemophilia is. Hemophilia is a blood does not properly coagulatedgeneticdisease.”< p>for decades, scientists have studied the use of a virus the direct delivery of the gene into the cells of the potential. Although it was recently successfully used a virus to successfully transport the healthy gene and relieve the patient’s symptoms, but people still can not control the viral gene is inserted into the cellular DNA of the location, which will increase the unwanted mutation risk.

however, the CRISPR/Cas9 gene editing technology has changed that. Although the CRISPR/Cas9 is not perfect, but compared to other gene editing techniques, it is a precisely edit the genome of method, and is user friendly. Scientists tell CRISPR/Cas9 in cells looking for what kind of target DNA fragments, and the use of which DNA sequences to replace them or what kind of DNA sequence is inserted into the target DNA fragment location. CRISPR/Cas9 challenge is to send it into the body of the correct position.

to this end, Curiel and his team will viral delivery methods with CRISPR/Cas9 combined to overcome both the technical limitations. They use an adenovirus as the delivery ofcarrier, and the use of CRISPR/Cas9 as a Navigator and editor, once the CRISPR/Cas9 reach the target position, the desired gene is inserted into the unlikely to cause the problem regions of the genome.

Curiel said,“We use the CRISPR/Cas9 will this virus targeting to be referred to as the Safe Harbor regions of the genome. These genomic regions are silenced by DNA sequence, does not exist in the more active DNA regions, and thus where this type of editing is not likely to do any harmful thing to do.”

In order to maximize the gene editing efficiency and sustained period, Curiel and his colleagues the use of adenovirus as the delivery ofcarrier. It has been confirmed as a cause of the common cold virus, adenovirus in gene transfer than other virus more efficient.

this study of the mice during the experiment appear to be healthy, but the adenovirus of the security issues still exist, and the CRISPR/Cas9 system may elicit an immune response problem also still exists. Curiel and his team is to further study the joint use of adenovirus delivery and CRISPR/Cas9 technology’s safety and effectiveness. Biological Valley Bioon.com

Published at Sun, 22 Apr 2018 12:51:22 +0000