#9: RESEARCH: CRISPR, Junk DNA, and Big Data

#9: RESEARCH: CRISPR, Junk DNA, and Big Data

Nearly a year after its designation by Science as a runner-up for Breakthrough Technology of the Year 2012, GEN made its own pronouncement on CRISPR (clustered, regularly interspaced short palindromic repeats) and CRISPR-associated (Cas) proteins, saying the technologies have come of age.

As GEN’s Patricia Fitzpatrick Diamond, Ph.D., reported in July, researchers believe that recent breakthroughs in understanding the mechanisms of CRISPR/Cas offer great potential for biotechnological applications and understanding evolutionary dynamics. Unlike other tools, CRISPRs are constructed from RNA—a cheaper and easier starting material. CRISPRs can be designed and customized to induce cuts at precise location in the genome, and can make nicks simultaneously at more than one genomic location, allowing researchers to look at the effects of combinations of mutations. Indeed, CRISPR’s gene editing methods, along with those of developing the gene editing methods of TALENs (transcription activator-like effector nucleases), are the basis of a new startup company founded in November by five pioneers in genome editing technology, Editas Medicine.

This year could be remembered as the year researchers finally figured out the value of “junk” DNA. Scientists at Sydney’s Centenary Institute reported in August that the 97% of human DNA long referred to as junk is actually a previously unknown mechanism for regulating the activity of genes, thus increasing human understanding of the way cells develop and pointing to new possibilities for therapy. In a study published in Cell, John Rasko, Ph.D., and a team including Centenary’s head of bioinformatics, William Ritchie, Ph.D., showed how particular white blood cells used noncoding DNA to regulate the activity of a group of genes that determines their shape and function.

Big data’s promise for lowering healthcare costs and advancing personalized medicine—McKinsey Global Institute estimates institutions could generate up to $100 billion in value annually by applying big data strategies—led Berg to join Mount Sinai’s Icahn School of Medicine in launching a pharmaceutical and diagnostic R&D partnership designed to leverage the power of multi-omic biology and data analytics.

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