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This past weekend, the gene-editing technique CRISPR achieved a historic milestone by becoming the first drug to be delivered systemically to a human body. To treat genetic illnesses, CRISPR, or clustered regularly interspaced short palindromic repeats, effectively chops genomes and slices DNA. After being given as an IV infusion, the latest breakthrough, the product of a trial between biotech major Regeneron and Boston-based startup Intellia Therapeutics, addressed a rare condition. Other uses of CRISPR technology had previously been limited to ex vivo therapy, in which cells are taken from the body and genetically modified in a laboratory before being reintroduced back into the body. Jennifer Doudna, the co-founder of Intellia and the winner of the 2020 Nobel Prize in Chemistry for her work on CRISPR gene editing, recently told CNBC that the technology’s evolution from the publication of her early work to clinical trials showing it to be effective in treating diseases in less than 10 years represents “one of the fastest rollouts I think of technology from the fundamentals.” “It’s largely because the technology comes at a time when there’s a lot of information about genomes, as well as a lot of demand for genome editing,” Doudna said at the CNBC Global Evolve Summit in mid-June. In terms of the future, Doudna outlined numerous obstacles and potential for CRISPR. CRISPR delivery is still a major hurdle. While technology has progressed, getting the altered molecules to the cells in the locations where they are needed remains a hurdle. “This is especially a concern in clinical medicine,” Doudna said, “where the ability to edit brain cells, heart cells, or muscle cells has enormous potential but we don’t have the tools to bring the editors into those cells right now.” “We have the editors; we just don’t know how to get them to where they need to go,” says the narrator. The early focus was on sickle cell anemia. Ex vivo therapy, in which removed cells are modified in a laboratory and then reintroduced into a patient, has been a big part of CRISPR’s success so far. Blood stem cells can be “harvested, edited, and then reintroduced to patients,” according to Doudna. Sickle cell anemia, which is passed down genetically and affects about 100,000 Americans, has been a particularly good target for the technology because it can be “harvested, edited, and then reintroduced to patients.” CRISPR uses have also been focused on genetic abnormalities of the eye, as Doudna explained, “it’s certainly easier to deliver to the eye than to other regions of the body.” It has also shown to be easier to deliver the altered cells to the liver thus far. “A liver is an organ that takes up molecules in the body naturally,” she explained. Any success in removing the more than 100 liver illnesses that exist could have a significant influence on Americans’ lives. According to the American Liver Foundation, at least 30 million Americans, or one in every ten, have some form of liver disease. Next, we’ll look at the brain, heart, and muscles. The next stage in CRISPR innovation, according to Doudna, will be to get those cells into other regions of the body, such as the brain, heart, and muscles. “There are some technologies that already permit some of this, such as the usage of various types of viruses or virus-like particles,” she added. “I’m optimistic about the innovation that will come in the next few years in this respect.” Treatment costs are a worry. However, as technology advances and scientists obtain the ability to target diseases across the body, Doudna believes that CRISPR technology will need to become more affordable in order to be “widely impactful.” According to Doudna, treating sickle cell disease with CRISPR therapy costs roughly $2 million per patient. “That is clearly not a price threshold at which most people who can benefit from it will be able to afford it,” she said. While fixing delivery issues may help cut prices, Doudna believes the medical sector must discover out how to “scale molecular production so that we can save expenses.” CRISPR-Cas9 in Agriculture Other industries may gain from the growth of CRISPR technology, with agriculture being one of the first to benefit. Rather than trying to solve genetic problems through breeding, which can take months to years, or current methods for genetically modifying crops, which have exploded in popularity in recent decades but require the insertion of biological material from other species, CRISPR technology can manipulate plant genes “without touching anything else,” according to Doudna. “This opens the door to a number of things that can be done today to both address climate change concerns, deal with drought conditions, and introduce features in plants that protect them from pests,” she added./nRead More

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