Hemophilia Cured in Mice
Gene therapy involves substituting or introducing a therapeutic gene to replace or accompany the defective one. Many previous attempts at gene therapy have utilized retroviruses, however the main pitfall of retroviruses is that scientists cannot control where the therapeutic gene will be inserted. This is a huge drawback as the therapeutic gene might be inserted in a potentially useful or necessary part of the genome, which could lead to even more genetic mutation. by ICSident ( CC License) via Wikimedia Recently, a team of scientists led by Dr Holmes and Dr High used a zinc finger nuclease to repair hemophilia in mice . Zinc finger nucleases don’t actually exist in nature and are engineered from a fusion of a Zinc finger domain and a nuclease. Zinc Finger domains are typically found in transcription factors (proteins which help to control the rate of transcription of RNA from DNA) because they recognize and interact with very specific sections of DNA. Nucleases on the other h