Gene Editing in Adult Mice

Researchers at the Massachusetts Institute of Technology have cured mice that had a single-gene mutation which caused a rare liver disease. Inserting DNA is a fairly simple task while making changes later in embryonic development proves to be much more difficult, especially editing an existing gene. Tyrosinemia is a rare, autosomal recessive disorder which affects the livers ability to break down the amino acid tyrosine. The disease is a single mutation that encodes the enzyme fumarylacetoacetate hydrolase (Fah).
DNA of bacteria have the ability to target and snip DNA of viruses; this was the system that was used in this research. Programmed cells were injected into adult mice were the Fah mutation was corrected. This has been done before in an embryonic stage of development but never in a full grown adult. This is the first successful gene editing done in an adult animal and proves to be a huge step for this research and gives hope that eventually this will be useful technique for humans.
Problems with this technique include getting a wrong portion of DNA cut which could cause extensive damage. Scientist are working was to safely and efficiently use this technique.

For more information on Tyrosinemia visit the Genetics Home Reference web page. 
Original Article:  Digital Journal