Genome Editing for R&D and Therapeutics
March 1, 2013 (Vol. 33, No. 5)
Kathy Liszewski

The ability to edit the human genome in a targeted and specific way allows correcting genes with harmful mutations, producing therapeutic proteins, and custom designing cell lines to model human diseases, such as cancer. Many feel that gene editing promises to write the next chapter in biomedicine.

RNA-Based Editing

Two major challenges with the introduction of zinc finger or TALEN nucleases are getting a high enough level of expression and persistence of the introduced DNA constructs, notes Matt Angel, Ph.D., CEO, Factor Bioscience. “The use of synthetic RNA to express proteins is often overlooked. Our company is developing research tools and therapeutics using our RNA-based gene-editing and reprogramming technologies.”

According to Dr. Angel, the idea is to deliver an RNA that encodes a gene-editing protein, such as a TALEN, that is targeted to a specific gene. “When our RiboSlice™ molecules are delivered, cells translate them into TALENs that subsequently create either nicks or double-strand breaks in the cell’s DNA. This leads to the disruption or targeted insertion of a specified gene if we co-deliver the desired gene DNA template.”