Novel technology vastly improves CRISPR/Cas9 accuracy

PHYS.org, November 18, 2015. http://phys.org/news/2015-11-technology-vastly-crisprcas9-accuracy.html

In an effort to improve CRISPR/Cas9 targeting research at the University of Massachusetts Medical School combined the old with the new. Zinc Finger DNA editing was originally developed in the early 1990’s by combining the DNA binding Zinc Finger protein domain with the Fok1 nuclease domain.  Since then researchers have developed tools to design zinc-finger binding domains to target specific DNA sequences.  The researchers at the University of Massachusetts combined this ability with the new CRISPR/Cas9 system by fusing a Zinc Finger domain to Cas9 to enhance the specificity of DNA targeting.  By combining these two different DNA targeting technologies the risks of off target effects.

‘Designer cells’ reverse one-year-old’s cancer

James Gallagher, BBC, November 5, 2015. http://www.bbc.com/news/health-34731498

Genetic therapy has reached a new milestone with one-year-old Layla seemingly cured of childhood leukemia.  Layla was recently diagnosed with incurable leukemia and as a last ditch effort her parents and doctors received permission to try an experimental gene therapy treatment.  Donor immune cells were edited with TALENs to seek out and kill only the leukemia cells in Layla’s body.  Only a few months after the treatment Layla had no traces of leukemia in her body.  While this treatment used an older, more expensive gene editing technique known as TALENs the advent of the cheaper and easier CRISPR/Cas9 technology may make more treatments like this a reality.

Editing of Pig DNA May Lead to More Organs for People

Carl Zimmer, New York Times, October 15, 2015. http://mobile.nytimes.com/2015/10/20/science/editing-of-pig-dna-may-lead-to-more-organs-for-people.html?_r=0

Each year thousands of people wait for a transplant organ.  Pig organs have long been thought of as a potentially limitless source of transplant organs but research was slowed in 1998 when scientists discovered that the pig genome contains viral DNA that can infect and alter human cells.  This obstacle may have recently been overcome when a single CRISPR/Cas9 system was used to alter 62 pig genes to deactivate this viral DNA.  With further research gene editing technology could finally create the limitless supply of transplant organs needed.

Who Owns CRISPR?

3Jenny Rood, The Scientist, April 3, 2015. http://www.the-scientist.com/?articles.view/articleNo/42595/title/Who-Owns-CRISPR-/.

The first US patent for CRISPR/Cas gene editing was awarded to Feng Zhang of the Broad Institute and MIT on April 15, 2014, however a second patent application was submitted seven months prior to Zhang’s by Jennifer Doudna of UC-Berkeley and Emmanuelle Charpentier of the Helmholtz Center for Infection Research in Germany.  Both Doudna/Charpentier and Zhang had been working on CRISPR/Cas systems with the controversy over who owns the CRISPR/Cas Intellectual property stemming from the first date both groups published.  Doudna was the first to demonstrate CRISPR/Cas systems ability to edit targeted DNA, however Zhang was the first to demonstrate that this technology can be used to edit human cells.  Since both groups have set up competing companies the fight over who owns this intellectual property could continue in the courts for many years.

New CRISPR “Cut-and-Paste” System Corrects Metabolic Liver Disease

Genetic and Engineering News 2 February, 2015 http://www.genengnews.com/gen-news-highlights/new-crispr-cut-and-paste-system-corrects-metabolic-liver-disease/81252314/

Scientists at the University of Pennsylvania have used an adeno-associated virus and CRISPR/Cas9 technology to correct a rare liver disease that results from a defect in the urea cycle.  Interestingly, the researchers found that the treatment was more effective in newborn over adult mice.  Further improvements of the efficiency of repaired DNA integration is needed before genetic treatments are available for humans.  The original research can be found at http://www.nature.com/nbt/journal/vaop/ncurrent/full/nbt.3469.html.

Editas Medicine raises $94.4 million in year’s first IPO

Robert Weisman, The Boston Globe, 2 February, 2015 https://www.bostonglobe.com/business/2016/02/02/editas-medicine-raises-million-year-first-ipo/cnusWaNwNdlGkPsF1QAGlL/story.html

Editas Medice is the latest CRISPR startup to go public and the first IPO in 2016.  The IPO sold 5.9 million shares at $16 each, bringing in $94.4 million.  Despite the promising IPO, the future of Editas may rest in the patent dispute currently playing out over CRISPR.  Editas has licensed the technology from the Broad Institute, which is currently in a patent battle with UC-Berkley with the results from the dispute expected in 1-2 years.

The UK Just Green-Lit CRISPR Gene Editing in Human Embryos

Ben Wiseman, Wired.com, 2 February, 2015 http://www.wired.com/2016/02/the-uk-just-green-lit-crispr-gene-editing-in-human-embryos/

The Human Fertilization and Embryology Authority in Britain have approved the use of CRISPR/Cas9 gene to edit viable human embryos.  The approval is only for a single research group investigating embryonic development.  Unlike in the US, in Britain all experiments using human embryos must be approved by the government, meaning this research will not lead to the routine editing of embryos.

A Call to Fight Malaria One Mosquito at a Time by Altering DNA

Carl Zimmer, New York Times, July 17, 2014. http://mobile.nytimes.com/2014/07/17/science/a-call-to-fight-malaria-one-mosquito-at-a-time-by-altering-dna.html

Worldwide 600,000 individuals die from malaria each year.  Currently the best defenses against malaria include destroying habitats where mosquitoes breed, the distribution of insect nets, and the spraying of insecticides.  Recently CRISPR gene editing technology has been used to insert genes into the mosquito genome that imparts resistance to the malaria parasite.  In addition scientist can also insert the CRISPR gene editing system needed to propagate these genes through an entire population.  This technique, coined a “Gene Drive,” could be used to make entire populations of mosquitoes resistance to malaria thus eliminating human infection.  This same technology could also be used to reverse herbicide resistance in weeds and antibiotic resistance in bacteria.