Gene Editing Control Using Amino Acids

July 9, 2018  Source: FierceBiotech 152

Gene editing and gene therapy can delete disease-causing genes in order to treat specific diseases. But the skill to control unwanted genes is a point of concern, as it might cause severe side effects. A research team from Cardiff University and the University of Bath have devised a switch to control protein expression in mouse embryos, which could be used in human gene therapies in future. 

The scientific team tested the genetically engineered amino acid switch, named BOC in mice. Under a UV light, the amino acid glows green. By genetic code expansion methodology, the targeted gene was selected using this fluorescence, then edited and developed into mice. Under BOC exposure, it won’t glow, whereas the non-exposed ones remain green. BOC is cheap and nontoxic and works in any protein in any species.

Modification in gene therapies are not a novel idea, but the present approaches have several key points that should be addressed before use in humans. For instance, a research team from King's College, are trying to treat spinal cord injuries using gene therapy with an antibiotic-based "off" switch. This action generates enzyme production that could heal scars and allow the neurons to regenerate. Doxycycline is delivered after gene therapy to trigger the "off" switch. A small portion of the target gene stays active even after the therapy is switched off. Hence, the team is working to switch the gene completely off and move to trials in larger animals. In future, the UK team is planning to study aging or enhance regenerative medicine using this gene-editing skill.

By Ddu