Next-Generation CRISPR Approaches

The discovery of the CRISPR-Cas9 system has ushered in a new era of cell and gene therapy. This revolutionary technology, adapted from a prokaryotic immune defense mechanism, enables precise manipulation of DNA sequences. By employing guide RNA (gRNA) molecules to direct the Cas9 nuclease to specific genomic loci, researchers can induce targeted double-stranded breaks, facilitating gene editing. This capability has been harnessed to modify eukaryotic cells, including induced pluripotent stem cells, hematopoietic stem cells, and immune cells such as CAR-T, NK, and NKT cells.

Translating CRISPR-based therapies from bench to bedside is a complex process requiring specialized expertise and resources. Collaborating with a contract development and manufacturing organization (CDMO) experienced in CRISPR technologies can streamline this journey. By providing essential components like nucleases, gRNAs, and HDR templates, a reliable CDMO facilitates a seamless transition from discovery research to clinical trials and beyond.