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A number of serious diseases are known to occur because of defects or mutations in our DNA.
Curing such diseases could in principle be carried out by rewriting the DNA to fix the mutated base pairs. Yet until recently, scientists have remained largely stymied in their attempts to directly modify genes in a living animal.
New gene-editing technique
Findings described in the March 30, 2014, issue of Nature Biotechnology, however, reveal that a novel gene-editing technique, known as CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), can be used successfully in mice to reverse disease symptoms for a liver defect known as type I tyrosinemia. In humans, this potentially fatal ailment affects about one in 100,000 people.