This discovery will appear soon now, because researchers can use a tool called CRISPR-Cas9 to publish their work.
This tool, usually abbreviated as CRISPR, was first proved to be able to cut DNA in 20 1 1 year. It consists of a close relative of a protein and a DNA called RNA. Scientists can use it to cut the DNA chain at a very precise position, so that they can remove the mutated part of the gene from the genetic material chain.
In the past year alone, researchers all over the world have published dozens of scientific papers, detailing the research results-some promising, some critical-using CRISPR to cut and replace unnecessary DNA to develop treatments for cancer, HIV, blindness, chronic pain, muscular dystrophy and Huntington's disease, just to name a few.
"Because of CRISPR, the speed of basic research discovery has exploded," said biochemist and CRISPR expert Sam Sternberg, who is the head of the technology development team of Caribou Biosciences Inc in Berkeley, California, which is developing medical, agricultural and biological research solutions based on CRISPR.
Although any treatment based on CRISPR will take several years to be tested on human, "Robert Jeffrey Sternberg said in numerous interviews with Daily Life Science, and the new publication outlined the new discoveries of human health and human genetics by using this new tool. Of course, humans are not the only species with a genome. CRISPR is also used in animals and plants, from eliminating parasites, such as those that cause malaria and Lyme disease, to increasing the production of potatoes, oranges and tomatoes.
The effect of [CRISPR] is incredible. This has brought a revolution to the daily life of most laboratories, "said Jason Serc, the principal researcher and molecular biologist of the Sheltz Laboratory in new york Cold Spring Harbor Laboratory. Sheltzer and his team are using CRISPR to understand the biology of chromosomes and how chromosome-related errors can lead to cancer.
Neville Sanjana, assistant professor of biology, neuroscience and physiology from new york Genome Center and new york University, said, "I very much hope that in the next decade, gene editing will be transformed from a major research tool to a tool that can be used for new treatments in clinic.
Here, let's take a look at the latest progress in the fight against 10 disease, which proves the ability of CRISPR and hints at the future.
Cancer (royaltystockphoto/Shutterstock) A method to treat cancer has hinted at human beings. Since 460-370 BC, the Greek doctor Hippocrates invented the word: karkinos. However, because cancer, like many diseases, is caused by mutations in the human genome, researchers say that treatment based on CRISPR may one day slow down the spread of tumors or completely reverse diseases.
China has done some early work in this field. Nature magazine reported that in places where laws and regulations on human gene editing are more relaxed than in the United States,
In 20 16 10, a lung cancer patient in China became the first person in the world to receive cell injection modified by CRISPR. Researchers led by Dr. Lu You, an oncologist at Chengdu Sichuan University, modified immune cells extracted from patients' own blood, and banned the gene that produces a protein, which is usually hijacked by cancer cells for division and reproduction. I hope that without this protein, cancer cells will not multiply and the immune system will beat the United States.
The research team is also looking for ways to use CRISPR to fight cancer. Dr Carl June, director of translational research at abramson Cancer Center, University of Pennsylvania, and his colleagues were approved by the National Institutes of Health in June, 20 16 to conduct clinical trials on 8 patients with advanced melanoma (skin cancer). According to the university statement, sarcoma (soft tissue cancer) and multiple myeloma (bone marrow cancer). In this clinical trial, researchers will use CRISPR to change three genes in patients' own immune system cells, hoping that these cells can destroy cancer cells in their bodies.
Hiv (sebastian Kaulitzki | Shutterstock) It has always been a difficult battle to eradicate the virus that causes AIDS. This virus not only infects immune cells that attack the virus in the human body, but also is a notorious mutation factor. After HIV hijacks a cell in the body and begins to replicate, it will produce many genetic variations of its own, which will help it avoid drug treatment. According to the World Health Organization, this drug resistance is a huge problem in the treatment of HIV-infected people.
CRISPR has included HIV in its sight. 2065438+May 2007, researchers from Temple University and University of Pittsburgh used CRISPR to intercept the virus from virus-infected cells and shut down the replication ability of the virus. Researchers led by Chen Liang, a virologist at McGill University, said that the use of this technology was tested in three different animal models, which was the first time that researchers showed a way to eliminate HIV from infected cells. They reported their findings in the journal Molecular Therapy.
Ralwel/Shutterstock About 30,000 people in the United States suffer from a genetic disease called Huntington's disease. According to the Huntington Dance Association of the United States, this fatal genetic disease will lead to brain nerve degeneration over time. Symptoms include personality changes, mood swings, unsteady gait and slow speech. "KDSP" and "KDSP" are caused by a defective gene, which is larger than normal and produces a protein called Huntington protein. This protein is larger than normal, and then it is broken down into smaller toxic fragments and accumulated in neurons, destroying its function. According to the National Institutes of Health,
However, from June 2065438 to June 2007, scientists reported in the Journal of Clinical Investigation that they had reversed the disease in laboratory mice, and these mice had been modified to replace their Huntington protein genes with human mutant Huntington protein genes. Post-doctoral researcher in the Department of Human Genetics at Emory University and Ren of the Institute of Genetics and Developmental Biology, Chinese Academy of Sciences used CRISPR to cut off some mutated Huntington genes and produce toxic sites. The toxic fragments in the mouse brain decreased and neurons began to heal. The affected mice recovered some motor control, balance and grip strength. Although they are not as good as healthy mice in some tasks, the results show that CRISPR is helpful to counter this situation. Scientists emphasize that more rigorous research is needed before this therapy can be applied to humans.
Chiccodic/Shutterstock Duchenne muscular dystrophy is one of the longest genes in human body because of a gene called dystrophin. A research team of Southwest Medical Center of the University of Texas led by Eric Olson, a professor of molecular biology, is working with CRISPR to find a way to fight against Duchenne muscular dystrophy (sps). "Because of the mutation of dystrophin gene, the human body has not formed functional dystrophin, which is very important for the health of muscle fibers. Over time, this lack of protein will lead to progressive muscle degeneration and weakness.
20 17 In April, Olson and his team reported in Science Progress that they used a variant of CRISPR tool called CRISPR-Cpf 1 to correct the mutation that caused Duchenne muscular dystrophy. They fixed the gene in human cells cultured in laboratory Petri dishes and mice carrying the defective gene.
CRISPR-Cpf 1 is another tool in the gene editing toolbox. According to the statement of Southwestern Medical Center in Utah, USA, it is different from the more commonly used CRISPR-Cas9 because it is smaller and therefore easier to transport to muscle cells. It also identified a DNA sequence different from Cas9, which is very useful for editing a long dystrophin gene.
Prevention of blindness (Hannah Botcher/Stoke. XCHNG) One of the most common causes of blindness in children is a disease called Leber congenital amaurosis, which affects about 2 to 3 people. According to the National Institutes of Health, there is one in every 654.38 million+newborns. This condition is hereditary and is caused by at least 14 gene mutation responsible for normal vision.
Editas, a biotechnology company in Cambridge, Massachusetts, is studying a treatment based on CRISPR to reverse a disease called Leber's congenital amaurosis 10. According to Xconomy, a biotechnology news website, the company plans to submit the necessary documents to the US Food and Drug Administration before the end of 20 17 to start the first human trial for this situation.
Editas was founded by Zhang Feng, a professor of bioengineering at MIT, who proved that CRISPR-Cas9 can be used in human cells. Jennifer Du DNA of the University of California at Berkeley and Emmanuel Chapentier of the University of Vienna at that time also proved that CRISPR-Cas9 can intercept DNA, and they applied for the patent of this technology on 20 12. Broad Institute, a subsidiary of Massachusetts Institute of Technology, filed a patent in April 20 14, followed up quickly, and finally obtained the patent. According to Nature magazine, from 2065438 to February 2007, the University of California at Berkeley filed a lawsuit, claiming that Doudna was the first one. Since then, the patents of Bode Institute have been supported.
Chronic pain (Stasique/Shutterstock) Chronic pain is not a hereditary disease, but scientists are studying how to use CRISPR to reduce inflammation by changing genes, thus inhibiting back and joint pain. Under normal circumstances, inflammation is the way the body tells the immune system to repair tissue. On the other hand, chronic inflammation can damage tissues and eventually lead to mild pain.
20 17 In March, a research team led by Robby Bowles, an assistant professor of bioengineering at the University of Utah, reported that they used CRISPR to prevent some cells from producing molecules, which were designed to break down tissues and lead to painful inflammation. According to the university statement,
This technique can be used to delay tissue degeneration after back surgery. This can accelerate healing and reduce the need for additional surgery to correct tissue damage.
Lyme disease (CDC) Kevin EsWei Erte, an evolutionary biologist at Massachusetts Institute of Technology, wants to eliminate Lyme disease, which is caused by tick-borne bacteria and can spread from deer ticks to humans. The Centers for Disease Control and Prevention said that if left untreated, this infection will lead to arthritis, neuralgia, palpitations, facial paralysis and other problems.
Although the bacteria causing Lyme disease were transmitted to humans by deer ticks, ticks did not have such bacteria themselves when they hatched from eggs. On the contrary, young ticks absorb bacteria when they eat, usually in white-footed mice. Wilt, the ace, has to be reduced. Wired said that the white-footed mice were modified with the CRISPR-Cas9 gene to make themselves and their offspring immune to bacteria, which would not be transmitted to ticks, thus causing diseases. From June 2065438 to June 2006, Esvelt introduced his solution to the residents of Nantucket Island and Martha's Vineyard Island in Massachusetts. According to the Cape Cod Times, this is the main problem of Lyme disease. However, these mice will not be released to the island until further tests are completed, which may take several years.
Malaria (James Ghassani). Provided by Paul I Center for Disease Control. ; Professor Frank Hadley Collins, malaria kills thousands of people every year. 20 15 is the latest year counted by the world health organization, with about 2120,000 malaria cases and 429,000 malaria deaths.
Having solved the problem at the source, the research team in Imperial College London, London is working to reduce the number of mosquitoes that transmit malaria. According to a statement of the college, a group of scientists led by Professor Austin Burt and Professor andre christie will study two major action plans: genetically modifying male mosquitoes to produce more male offspring. The research team reported in Nature that from 20 15 to 12, they found three genes that reduced the fertility of female mosquitoes. They also announced that they found that CRISPR could target at least one of them.
Crops (Linda &; Just as CRISPR can be used to modify the genomes of humans and animals, it can also be used to modify the genomes of plants. Scientists are studying how to use the gene editing ability of this tool to reduce diseases of some crops and make others more robust.
Sophien Kamoun, a professor at Norwich sainsbury Laboratory in Britain, is studying how to remove genes that make potatoes and wheat susceptible to diseases. PhysOrg report. According to Nature magazine, Zachary Lippman, a geneticist in new york Cold Spring Harbor Laboratory, is using CRISPR technology to develop a tomato plant whose branches are optimized to bear the weight of mature tomatoes without breaking. According to Nature News, in California, several laboratories are trying to use CRISPR to treat a plant disease called citrus greening, which is caused by bacteria spread by insects flying between plants in citrus forests.
A viable human embryo has been edited, and the speed from hypothesis to result based on CRISPR is shocking. Serc told Life Science that experiments that used to take several months now only take a few weeks. This speed has aroused some concerns of policy makers and stakeholders, especially when this technology is used in humans.
2065438+In February 2007, scientists from the National Academy of Science, Engineering and Medical Sciences released an evaluation report on human gene editing, saying that it was acceptable, but only under certain conditions. According to Science News, the organization also said that it is ethically permissible to change the cells in embryos, eggs and * * *, provided that it is done to correct diseases or disabilities, not to enhance a person's appearance or ability.
Although no scientists in the United States have used CRISPR to modify a viable human embryo, the research team led by Liu Jianqiao of Guangzhou Medical University in China reported this progress in the March 201July issue of Molecular Genetics and Genomics. Scientists use CRISPR-Cas9 gene to introduce and edit the pathogenic mutation of human embryos. This study shows that gene editing can be done in the embryonic stage. These embryos are not implanted in the human body.
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