Gene therapy comes of age: We can now edit entire genomes to cure diseases

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For a long time, people thought HIV was incurable. The main reason was that HIV is a retrovirus, meaning that it inserts its own viral DNA into the genome of its host - perhaps we could treat the symptoms of HIV, but many doubted it was possible to actually correct the genes themselves. Our techniques for slicing up DNA are very advanced when that DNA sits suspended in a test solution, but nearly useless when we need to accurately edit millions of copies of a gene spread throughout a complex, living animal. Technologies aimed at addressing that problem have been the topic of intense study in recent years, and this week MIT announced that one of the most promising lines of research has achieved its first major goal: researchers have permanently cured a genetic disease in an adult animal.

This is a proof of concept for something medicine has been teasing for decades: useful, whole-body genome editing in fully developed adults. Until recently, most such manipulation was possible only during early development - and many genetic diseases don't make themselves known until after birth, or even much later in life. While breakthroughs in whole-genome sequencing are bringing genetic early-warning to a whole new level for parents, there are still plenty of ways to acquire problem DNA later in life - most notably, through viruses like HIV. Whether we're talking about a hereditary genetic disease like Alzheimer's or an acquired one like radiation damage, MIT's newest breakthrough has the potential to help.