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Today, medicine recognizes more than 5,000 genetic diseases caused by mistakes in DNA, and the majority of these illnesses have no cure or treatments. These are the diseases that stand to benefit most from genomic medicine, and specifically, the newest and most powerful genome-editing technology, called CRISPR.
CRISPR has taken the research community by storm, because it can make DNA changes in many different settings and many different kinds of cells. Scientists can now much more rapidly and comprehensively investigate what different genes do and how they work together.
The technology is young, and it will take time to fully realize its promise and develop medicines using the technique. Some diseases are more challenging than others, and there is a lot of work to be done to extend CRISPR’s capabilities.
Scientists have arrived at a watershed moment in their understanding of genomic science. Not only have researchers identified many of the mutations that cause a variety of diseases, but now there is also a technology that could create new medicines that directly target and correct those mutations.
To realize the potential of treating genetically driven diseases with medicines based on CRISPR, the challenge today is to safely and systematically advance the technology to the next phases of testing and ultimately into studies in people.
Read full, original post: What If Doctors Could Heal Broken Genes?