Genetic Engineering & Biotechnology News

DEC 2017

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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18 | DECEMBER 2017 | ➜ Kate Marusina, Ph.D. Tricks of the genome-writing trade were freely exchanged at the first an- nual Genome Writers Guild confer- ence entitled, "Realizing the Future: Genome Engineering," which was held at the University of Minnesota in July 2017. Besides suggesting ways to release genome-writing creativity, speakers at the meeting discussed technical advances, potential biotechnology ap- plications, and prospects for clinical translation. The meeting even included TED-style talks and a science fiction writer's views on genome engineering's utopian (and dystopian) possibilities. The bulk of the meeting, however, focused on more immediate techni- cal issues. Most sessions focused on the latest genome-engineering tools and their diverse applications, from dissecting the cell's molecular mecha- nisms, to harnessing these mecha- nisms for whatever purposes that are deemed worthy. Genome editing is giving us the power to change the world, a power that needs to be wielded responsibly. For example, therapeutic and repro- ductive genome editing have tremen- dous potential to alleviate illness, but we need to ask if this potential be realized safely, without unintended consequences. High-Fidelity CRISPR Toolbox "CRISPR gene-editing technology is taking biology by storm," points Mark Behlke, M.D., Ph.D, chief sci- entific officer, Integrated DNA Tech- nologies (IDT). "The CRISPR toolbox consists of three primary components: a guide RNA, which targets a specific locus in the gene; a Cas9 nuclease, which creates a DNA break at the locus; and a DNA repair template, which enables gene replacements and insertions. IDT holistically optimized the entire toolbox, resulting in a high- ly efficient, error-resistant system." While there are numerous ap- proaches to implementation of CRIS- PR/Cas9 gene editing, many of them experience a relatively high rate of off-target effects, limiting the system's applicability. Dr. Behlke explains that if Cas9 is expressed from DNA or even messenger RNA, the enzyme persists in a cell for a long time, increasing chanc- es of cleavage at nonspecific sites. IDT and others have demonstrated that a ribonucleoprotein (RNP) com- plex consisting of Cas9 and guide RNA could be delivered at high con- centration, but degrades rather quick- ly. "The RNP approach limits the amount of catalytic components and gives [a] "fast-on/fast-off" character to the genome-editing machinery," says Using CRISPR to Improve Disease Modeling B E S T O F C R I S P R 2 017 Genome Engineering: Many Ways to Beat Genome Writer's Block Figure 1. To address concerns about the off-target editing commonly observed in CRISPR/Cas9 applications, Integrated DNA Technologies has developed a high-fidelity, Streptococcus pyogenes Cas9 nuclease. When delivered as a ribonucleoprotein, this nuclease, called Alt-R® S.p. HiFi Cas9 Nuclease, can provide superior on-target performance, maintaining the high potency of wild-type Cas9 while reducing off-target editing.

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