CRISPR技术,遗传性失明 z-bo 2020-09-21 416

　　CRISPR technology is a system suitable for bacteria that can resist invading viruses. The bacterium first copies the genetic code of the invader into a special sequence of ribonucleic acid (RNA). When the virus returns, the RNA binds to a protein called Cas9 and directs it to match the viral genes. Protein can suppress genes. By modifying the system, scientists can program Cas9 to turn on or off selected genes or rewrite their genetic code.

　　In this study, researchers at Cedars-Sinai Medical Center designed a CRISPR/Cas9 system to eliminate genetic mutations that cause the loss of photoreceptor cells in the eye. They injected the system into young experimental rats that have been modified to mimic hereditary retinitis pigmentosa, called autosomal dominant inheritance, which contains mutations in this gene. By measuring the photodynamic reflex, which involves moving the head in response to stripes of different brightness after a single injection, the researchers can improve the vision of these rats compared to the control group. I have found.

　　Dr. Clive Svendsen, co-author of the

　　study, said that by modifying the components of the CRISPR/Cas9 system and using new viral vector technology, the validity and consistency of these results can be improved. He believes that in future further research, reliable genome editing of the system can provide a means to correct various genetic diseases.

　　The Governor’s Institute of Regenerative Medicine said: Clinical transformation has paved the way.