Apply gene editing technology in treatment of retinitis pigmentosa

US scientists have been using gene editing repair cells may lead to mutations pigmentary retinal degeneration, this genetic disease can lead to retinal degeneration, and within a decade lead to total blindness. In fact, this disease is one of the main causes of the loss of young people from around the world. The researchers used CRISPR technique to repair the affected cells, which marks the first time ever, scientists have replaced the stem cells from the patient's own tissue and sensory disorders associated defective gene.

The results of this study were published in Scientific Reports, the paper details how the researchers extracted from the skin of patients with retinitis pigmentosa in the sample. Then use skin samples, researchers in the laboratory to create stem cells used as a basis for gene editing. CRISPR technology allows scientists to cut and replace the individual GMOs DNA, effectively rewriting the genetic code.

In this case, the researchers used the technique to repair CRISPR stem cells from a patient (still contain lead pigmentary retinal degeneration mutation) in a gene defect. CRISPR is not allowed because the current technology is also applied to human beings, so this study will only go so far; but here the achievements show that the future is expected to be used in human gene editing.

According to the researchers, if the repaired stem cells into healthy retinal cells, they can be transplanted back into the patient's body. Without mutation, healthy cells can make patients see again. "Our goal is to develop a personalized treatment of eye diseases," Columbia University Medical Center ophthalmologist Stephen Tsang said, "We still have some way to go, but we believe that the first therapeutic application is the treatment of CRISPR eye disease. Here we have shown that the initial step is feasible."

Traditional organ transplant is different because the repair cells from the patient's own body tissue. The researchers believe that the patient's body will not be rejection of the immune system, and it is easy to receive genetically edit cells. This means there is no need to use drugs to inhibit organ rejection. The researchers said the most likely candidate is the first clinical application of CRISPR. Compared to other parts of the body, eye surgery easier and more easily detected after surgery. "Retinal disease is a perfect model of dry cell surgery, because we have advanced surgical techniques, the cells can be accurately implanted to where it is needed," Vinit Mahajan, team member from the University of Iowa said.

Due to genetic problems also edit applied to human security and moral, we do not know when we can use CRISPR to treat blindness. But scientists involved in the study believe that their proof of concept demonstrated significant potential of this technology, and we should go this way. Tsang said, "There is a lot of work to do before we treat patients. We should ensure that we only modify specific single mutation, without other changes in the genome."

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