That's the super exciting and interesting part of literally today. There are two new technologies that are about to combine that will probably get us to "reliab

That's the super exciting and interesting part of literally today. There are two new technologies that are about to combine that will probably get us to "reliably fix single nucleotide mutations." in a platform sense within the next 5 years. The first technology is the production and delivery of messenger RNA made synthetically, and the second is the fusion of the recognition portion of CRISPR with a class of enzymes known as base editors. Of course we'll have to start with the liver and with diseases that move the needle (i.e., not N of 1), but once the technology is approved in this case things get a lot more interesting for other genetic diseases that don't move the needle. Tim Yu developing the ASO for Mila is the perfect example of how once the technology is working for a money-making indication, less profitable treatments can follow. If you want to read more about this, check out Beam Therapeutics, Moderna Therapeutics, Intellia Therapeutics, CRISPR Therapeutics, Editas, STRAND Therapeutics, Translate.bio, BioNTech, Josh Rosenthal at Woods Hole, Feng Zheng at MIT/Broad, and David Liu at Harvard.