|Gene Repair Offers Hope and Challenges
Many rare disorders originate from gene mutations— typos in the DNA that disrupt normal cellular functions. What if it were possible to fix errors in DNA sequences that cause genetic diseases? Would this permanently cure the disease? Can genes be mended as easily as jeans? Not yet, but interestingly, the concept is not beyond the realm of possibility.
In recent years, scientists have learned how to manipulate a precise DNA cutting mechanism found in bacteria called CRISPR. This system is used by microorganisms to defend themselves from foreign DNA by comparing invading sequences to those stored on a genetic “wanted list." If there’s a match, the DNA is cut. By co-opting this bacterial machinery, DNA sequences of any type, including those of disease-causing genes, can be cut. Once cut, DNA repair mechanisms can be harnessed to insert a corrected sequence and the genetic patch is completed.