Self-focusing therapeutic gene delivery with intelligent gene vector swarms: Intra-swarm signalling through receptor transgene expression in targeted cells

Conclusions It is hoped that proposed self-focusing cell-targeted gene vector swarms with receptor-mediated intra-swarm signalling could be particularly effective in ‘top-up’ gene delivery scenarios, achieving high-level and sustained expression of therapeutic transgenes that are prone to shut-down through degradation and silencing. Crucially, in contrast to low-precision ‘general location’ vector guidance by diffusible chemo-attractants, ear-marking non-diffusible receptors can provide high-accuracy targeting of therapeutic vector particles to the specific cell, which has undergone a ‘successful cell-specific hit’ by a ‘scout’ vector particle. By employing such self-focusing gene vectors for improvement of cell-targeted delivery of therapeutic genes, e.g., in cancer therapy or gene addition therapy of recessive genetic diseases, it could be possible to broaden a leeway for the reduction of the vector load and, consequently, to minimize undesired vector cytotoxity, immune reactions, and the risk of inadvertent genetic modification of germline cells in genetic treatment in vivo.
Source: Artificial Intelligence in Medicine - Category: Bioinformatics Source Type: research