OFweek Medical Network January 12: According to a study by the Cedars-Sinai Medical Center in the United States, it is the first time that a new technology can be used to treat hereditary diseases by removing genetic defects and prevent rats with a hereditary blindness. Retinal degeneration.
A research team at the Governors Institute of Regenerative Medicine at Cedars-Sinai Medical Center, which focuses on hereditary retinitis pigmentosa, which can lead to blindness, has no cure. The researchers used a technique called CRISPR/Cas9 to remove a genetic mutation that causes blindness. CRISPR/Cas9 is adapted from a strategy used by bacteria to combat invading viruses. Although the study used rats, the results of this study are an important milestone because of its potential impact on humans.
The results of the study were published in Molecular Therapy. The study's senior author, the eye project research scientist and associate professor of biomedical science, Shaomei Wang, said: "Our data shows that with further development, we can Using this gene editing technology to treat hereditary retinitis pigmentosa in patients." Dr. Wang Shaomei graduated from Liaoning Medical University in early years, obtained a master's degree from China Medical University, and received a Ph.D. from Sheffield University, USA, focusing on retinitis pigmentosa and optic nerve. Cellular therapy of lesions, mechanisms of stem cell repair of vision, and immune responses of human cells in the eyes of animal models.
According to the National Institutes of Health, patients with retinitis pigmentosa develop night blindness in the early stages of the disease, along with retinal atrophy and pigmentation, reduced visual field, and ultimately blindness. Although generally rare, it is the most common hereditary retinal disease affecting approximately one in 4,000 people in the United States and Europe. In less than five years, the scientists of the CRISPR/Cas9-Cedars-Sinai Medical Center used to target retinitis pigmentosa have been used by genetic researchers. It has made the genome editing process easier, more reliable, and cheaper, and has revolutionized the science of genetic editing.
This technology is a system adapted from bacteria to fight invading viruses. The bacterium first copies the invader's genetic code into a special sequence of ribonucleic acid (RNA). When the virus returns, the RNA binds to a protein called Cas9, directing it to match the viral gene. This protein can inhibit this gene. By modifying this system, scientists can program Cas9 to turn the selected genes on or off, or to rewrite the genetic code.
In this study, researchers at the Cedars-Sinai Medical Center designed a CRISPR/Cas9 system to remove a genetic mutation that causes photoreceptor cell loss in the eye. They injected the system into young laboratory rats that have been engineered to mimic a hereditary retinitis pigmentosa, called autosomal dominant inheritance, which involves mutations in the gene. After a single injection, the visual acuity was measured by visual reflex measurements, including rotating the head in response to movement of different brightness stripes. The researchers found that the visual acuity of these rats became better compared to the control animals.
Dr. Clive Svendsen, co-author of the study, said the validity and consistency of these results could be improved by modifying the components of the CRISPR/Cas9 system and using new viral vector technologies. He believes that in the future, through more research, reliable genome editing through this system can provide a means to correct various genetic diseases.
Svendsen, director of the Regulatory Institute of Regenerative Medicine, said: "This is the first time that the CRISPR/Cas9 gene editor has been used to prevent vision loss in living animals. This is indeed a remarkable result for more exciting research and future clinical transformation. , paved the way."
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