Holiday Reading for a CRISPR Autumn Day

By Bio-IT World Staff 

 

November 26, 2014 | Holiday greetings from Boston, where we really are bracing for a crisper Thanksgiving as Winter Storm Cato bears down! While we’re out of the office, please enjoy some reading on the rapid rise of CRISPR gene editing, which the biotech industry has every reason to be thankful for this year. Most recently, Novartis and Atlas Venture invested $15 million in a new startup working on human therapies based on the new gene editing technology. Intellia Therapeutics, based in Cambridge, Mass., is the third such company to sprout up in the last twelve months, joining Editas Medicine right next door in Cambridge, and CRISPR Therapeutics, which operates out of Basel and London.

 

Intellia can’t claim the illustrious heritage of its counterparts: the co-discoverers of CRISPR’s gene editing powers, Jennifer Doudna and Emmanuelle Charpentier, are scientific founders of Editas and CRISPR Therapeutics, respectively. However, Intellia has attracted major talent from the biotech drug industry, with much of the executive team coming from companies and divisions that have overseen successful drug launches. Like CRISPR Therapeutics, Intellia plans to go after ex vivo applications first, modifying human immune cells outside the body to create personally tailored therapies for diseases like cancer. (Editas has so far been cagier about its disease applications.)

 

Lots more has gone on with CRISPR over the past year. Here is a selection of our coverage on this remarkably adaptable gene engineering system.

 

Breakthrough Prizes Highlight CRISPR Gene Editing, Gene Regulation: Jennifer Doudna and Emmanuelle Charpentier were big winners at this year’s Breakthrough Prizes, the multi-million-dollar scientific awards hosted in Silicon Valley by a coalition of tech titans.

 

CRISPR Knockin Mice Enable Rapid Gene Editing: Highlighting how adaptable the CRISPR-Cas9 system is, Feng Zhang of the Broad Institute and colleagues have engineered a strain of lab mice that naturally produce Cas9. That means scientists who want to knock out any gene in mice can quickly alter their gene expression with little more than some targeted RNA.

 

Could CRISPR Transform Wild Ecosystems?: George Church’s lab is already thinking ahead to environmental uses of CRISPR. If this technology is one day used to manipulate wild populations, like disease-carrying mosquitoes, we’ll need policies in place to clarify responsible procedures.

 

Advances in Genetic Engineering at the 2014 American Society for Microbiology Meeting: CRISPR was discovered as an antiviral system in bacteria, so it’s no surprise that it’s a hot topic in microbiology. CRISPR-Cas9 systems could even be used as highly specific antibiotics, cutting away harmful genes while leaving friendly microbes alone.

 

Broad Claims Patent on CRISPR Technology: While many of the early pioneers of CRISPR gene editing have made important commitments to open science, we can expect patent disputes in the future as this powerful technology becomes a practical tool.

 

A Crisper Image of Cas9 Structure: Even as scientists tinker with the basic structure of the Cas9 protein, it remains unclear exactly how this molecule binds to and cuts specific stretches of double-stranded DNA.

 

Gene Therapy’s Next Generation: Bio-IT World speaks to Feng Zhang and Keith Joung, scientific founders of Editas Medicine, about CRISPR’s potential as a therapeutic and the many challenges to adapting it for safe use in humans.