AMRITA Project: In vivo genomic engineering

Re-engineering adult animals and humans to overcome aging requires safe and efficient tools to allow introduction of synthetic biology circuits and programs.  The simplicity of CAS9/CRISPR to introduce DNA into defined chromosomal locations, delete unwanted DNA, create artificial transcription factors and inhibitors, as well as directed chromatin remodeling complexes promises a revolution in genomic engineering and synthetic biology.  Ex vivo manipulation is now easy and could be used to create personal designer cells to be reintroduced in vivo.  True in vivo genome engineering requires efficient cell-targeted delivery of CAS9/CRISPR and their derivatives.  As part of the AMRITA project, we are creating modified AAV (adeno-associated virus) gene therapy vectors designed to express CAS9/CRISPR and guide RNA to advance in vivo genomic engineering for rejuveneering.