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Of the many patients who need an organ from a donor, 90% go without. About 240 million people live with rare genetic diseases, most of which cannot be treated. Each year poor diets cause more than 10 million early deaths. Suffering on such an immense scale can appear hopeless. However, a technique called CRISPR gene editing promises to help deal with these issues and many more and proper regulation can help it develop.
CRISPR is like an editor for DNA. It can rewrite DNA, removing harmful mutations (突变) or adding protective ones. This summer, clinical trials (试验) will start on pig organs edited for human transplants. Last year, the first new treatment went on the market. It seemingly cures sickle-cell
disease and beta-thalassemia, two blood disorders that affect millions. If ongoing trials succeed, a one - time treatment might protect against heart attacks for life. CRISPR can also help farming. It can increase outputs or protect crops from climate change. Soon, consumers may get healthier,
tastier foods.
But now is a crucial time. Since its discovery in 2012, CRISPR has replaced old, less - effective ideas. Gene treatment, which uses viruses to insert genes, can treat rare genetic diseases but is expensive. Genetically modified (GM) crops, which borrow genes from other species, face opposition in Europe. CRISPR offers a new way. But to succeed, it needs continuous investment, which means achieving real-world successes. For this to happen, scientists must show they can get CRISPR into more body cells easily and cheaply. If it can create personalized treatments for individual mutations, it will be even more useful. This requires new science and better regulation.
Current regulations for rare - disease drugs aren't suitable for new medicines. They stop patients from getting new treatments. Developing drugs for small groups has always been hard, and
many CRISPR companies are struggling. But CRISPR is programmable, so the same drug can target different mutations. If safety testing and manufacturing standards are loosened, small - batch drugs for rare diseases can be made more cheaply. For patients who may die before drug approval, this is a good trade-off.
Agriculture also needs reform. In many regions , gene - edited foods are regulated like GM foods, though they're different. Gene - edited plants have their own genes adjusted, not genes from
other species. Britain plans to pass new, looser laws for gene - edited foods to address climate change threats to food security. But public trust in regulators and scientists could be a problem.