Daniel Cressey in Nature:
FENG ZHANG: DNA’s master editor: Borrowing from bacteria, a biologist helps to create a powerful tool for customizing DNA. With a nip here and a tuck there, a DNA-cutting mechanism that bacteria use to protect themselves from viruses became one of the hottest topics in biomedical research in 2013. And a young neuroscientist with a penchant for developing tools helped to make it happen. Thirty-two-year-old Feng Zhang of the Massachusetts Institute of Technology in Cambridge is among those leading the charge in using a system called CRISPR/Cas to edit genomes cheaply, easily and precisely. In January, his group showed that the system works in eukaryotic cells — ones with membrane-bound nuclei, including those of all animals and plants. This confirmed its potential for tweaking the genomes of mice, rats and even primates to aid research, improve human-disease modelling and develop treatments (L. Cong et al. Science 339, 819–823; 2013). But as hot as the story has been this year, “the CRISPR craze is likely just starting”, says Rodolphe Barrangou, a microbiologist at North Carolina State University in Raleigh. CRISPRs (clustered regularly interspaced palindromic repeats) are DNA sequences that many bacteria and archaea use to defend themselves. They encode RNAs that can specifically recognize a target sequence in a viral genome. The RNAs work in complex with a CRISPR-associated protein, or Cas, which snips the DNA of the invader.
…Zhang now says that he feels challenged to be creative with other applications. One particularly ambitious project on his slate is to build a library of CRISPRs that can delete any sequence in an organism’s entire genome in 100–200 base-pair increments. This could make it easier to investigate the function of non-coding DNA. But he is most interested in using CRISPR to treat neuropsychiatric conditions such as Huntington’s disease and schizophrenia by repairing genes in human tissues. To pursue therapeutic use of the technology, he and other CRISPR pioneers last month launched a company called Editas Medicine, based in Cambridge, that is backed by US$43 million in venture-capital funding. CRISPR “allows us to start to make corrections in the genome”, says Zhang. “Because it’s easy to program, it will open up the door to addressing mutations that affect few people but are very devastating.”
More here. (Note: I am convinced that CRISPR is one of the most important discoveries of the century)
