The "Maximally Modifiable Mouse" (Buck Institute for Research on Aging)
Many components of SENS require the addition of new genes to the genome of cells in the body. This technology, known as somatic gene therapy, has been a goal of medicine for decades, but it is still very far from perfect. Difficulty in controlling the precise location of gene insertion is a significant barrier to these efforts, as insertion in the "wrong" location could result in the cell becoming cancerous. To mitigate this difficulty, we have devised a new method for inserting genes into a pre-defined location. In the clinic, this will be done as a two-step process, in which first CRISPR is used to create a "landing pad" for the gene, and then the gene is inserted using an enzyme that only recognizes the landing pad. We have created mice that already have the landing pad, and now we plan to evaluate how well the insertion step works in different tissues.