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1st of many steps in the gene editing oversight.


PRINCETON, N.J., March 29, 2016 /PRNewswire/ — WIRB-Copernicus Group® (WCG™), one of the world’s leading providers of solutions that measurably improve the quality and efficiency of clinical research, today announced that it has assembled a team of world-renowned experts to advise the company regarding the latest advances in gene therapy research. The WCG Gene Therapy™ Advisory Board will convene today in Princeton, NJ.

“Human gene therapy is one of the fastest-growing areas of medical research, and also one of the most promising,” said WCG Chairman and Chief Executive Officer Donald A. Deieso, Ph.D. “The advances made by scientists and clinicians in the field of gene therapy have enabled us to target disease at the genetic level, redefining the concept of precision medicine.” He added, “More than that, gene transfer researchers have succeeded – over the course of a single lifetime – in transforming the world’s most persistent and lethal viruses into disease-fighting allies in the quest to improve human health.”

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Synthetic biology involves creating or re-engineering microbes or other organisms to perform specific tasks, like fighting obesity, monitoring chemical threats or creating biofuels. Essentially, biologists program single-celled organisms like bacteria and yeast much the same way one would program and control a robot.

But 10 years ago, it was extremely challenging to take a DNA sequence designed on a computer and turn it into a polymer that could implement its task in a specific host, say a mouse or human cell. Now, thanks to a multitude of innovations across computing, engineering, biology and other fields, researchers can type out any DNA sequence they want, email it to a synthesis company, and receive their completed DNA construct in a week. You can build entire chromosomes and entire genomes of bacteria in this way.

“Biology is the most powerful substrate for engineering that we know of,” said Christopher Voigt, Professor of Biological Engineering at MIT. “It’s more powerful than electrical engineering, mechanical engineering, materials science and others. Unlike all the other fields, we can look at what biology is already able to do. When we look at the natural world, we see things like the brain. That’s a complex place computing, electrical engineering and computer science can’t reach. The brain even constructs nanostructures very deliberately, something materials science has not accomplished.”

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(Photo credit: AP Photo/Jack Plunkett, File)

Astro Teller is tired of the paranoia surrounding artificial intelligence and robotics. The famous computer scientist’s sensitivity around the topic may be understandable considering he bears the brunt of some of that skepticism as the head of X, the Alphabet (and formerly Google) moonshot factory working on many of the company’s futuristic AI and robotics projects.

This past weekend, Teller, whose official title is “captain of moonshots,” took to the stage at the inaugural Silicon Valley Comic Con hoping to dispel some of these misconceptions around AI. His physician wife, Danielle Teller, presented alongside him on some of the fear mongering associated with genetic engineering in humans. After their presentation, the Tellers sat down with FORBES to go deeper on the issue to explain what they hoped to accomplish with their talk.

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WIKIMEDIA, ROBINSON RCRISPR-a bacterial immune response best known for its genome-editing applications in the lab-has yet again been adapted for scientific purposes, this time to track RNA within cells. Considering the case of synapses — the proteins required for these neural connections are produced from RNAs located at these contacts.

“Just as CRISPR-Cas9 is making genetic engineering accessible to any scientist with access to basic equipment, RNA-targeted Cas9 may support countless other efforts for studying the role of RNA processing in disease or for identifying drugs that reverse defects in RNA processing”, study coauthor David Nelles of the University of California, San Diego, said in a press release. Defective RNA transport is linked to a host of conditions ranging from autism to cancer and researchers need ways to measure RNA movement in order to develop treatments for these conditions. “Our current work focuses on tracking the movement of RNA inside the cell, but future developments could enable researchers to measure other RNA features or advance therapeutic approaches to correct disease-causing RNA behaviors”. But, Gene Yeo, Associate Professor of Cellular and Molecular Medicine at UC San Diego, and his team have applied the technique as a flexible means to targeting RNA in live cells.

Jennifer Doudna, the creator of the CRISPR-Cas9 system for DNA editing, also works out of the University of California research system, and is listed as a co-author for this study. A guide RNA, along with the addition of an oligonucleotide sequence, sent the Cas9 RNA-ward.

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This could have some truly profound implications for the treatment of all viruses, including HIV!


Researchers from Temple University have used the CRISPR/Cas9 gene editing tool to clear out the entire HIV-1 genome from a patient’s infected immune cells. It’s a remarkable achievement that could have profound implications for the treatment of AIDS and other retroviruses.

When we think about CRISPR/Cas9 we tend to think of it as a tool to eliminate heritable genetic diseases, or as a way to introduce new genes altogether. But as this new research shows, it also holds great promise as a means to eliminate viruses that have planted their nefarious genetic codes within host cells. This latest achievement now appears in Nature Scientific Reports.

Retroviruses, unlike regular run-of-the-mill viruses, insert copies of their genomes into host cells in order to replicate. Antiretroviral drugs have proven effective at controlling HIV after infection, but patients who stop taking these drugs suffer a quick relapse. Once treatment stops, the HIV reasserts itself, weakening the immune system, thus triggering the onset of acquired immune deficiency syndrome, or AIDS.

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It sounds really obvious, but hospitals aren’t for healthy people. The world’s entire health system is really there to react once people get ill. If doctors are able to catch an illness at stage one that’s great, but if it reaches stage three or four there’s often not that much that can be done. So what if we could treat patients at stage zero and predict the likelihood of contracting diseases? We could then get treatment to people who need it much earlier and take preventative steps to avoid illness altogether.

Currently, when we think of monitoring in healthcare we’re usually referring to monitoring patients’ reactions to drugs or treatments, but this is changing. No amateur runner’s uniform is complete these days without a Fitbit or some kind of analytics tool to monitor progress, so the idea of monitoring the healthy is becoming ingrained in the public’s consciousness. But Fitbits only scrape the surface of what we can do. What if the data from fitness trackers could be combined with medical records, census data and the details of supermarket loyalty cards to predict the likelihood of contracting a particular disease?

With big data we can move from reacting to predicting, but how do we move beyond just making predictions; how do we prevent disease from occurring altogether? Up until now all of our monitoring technology has been located outside of the body, but nano-sized entities made of DNA could one day patrol the body, only acting when they come into contact with specific cells – cancer cells, for example. The technology that would turn tiny machines – roughly the size of a virus – into molecular delivery trucks that transport medication is already being worked on by bioengineers. If this kind of technology can be used to treat cancer, without needing to release toxic agents into the body, can the same technology be inserted into a healthy person and lie in wait for the opportunity to fight disease on its host’s behalf?

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Making the most of the low light in the muddy rivers where it swims, the elephant nose fish survives by being able to spot predators amongst the muck with a uniquely shaped retina, the part of the eye that captures light. In a new study, researchers looked to the fish’s retinal structure to inform the design of a contact lens that can adjust its focus.

Imagine a that autofocuses within milliseconds. That could be life-changing for people with presbyopia, a stiffening of the eye’s that makes it difficult to focus on close objects. Presbyopia affects more than 1 billion people worldwide, half of whom do not have adequate correction, said the project’s leader, Hongrui Jiang, Ph.D., of the University of Wisconsin, Madison. And while glasses, conventional contact lenses and surgery provide some improvement, these options all involve the loss of contrast and sensitivity, as well as difficulty with night vision. Jiang’s idea is to design contacts that continuously adjust in concert with one’s own cornea and lens to recapture a person’s youthful vision.

The project, for which Jiang received a 2011 NIH Director’s New Innovator Award (an initiative of the NIH Common Fund) funded by the National Eye Institute, requires overcoming several engineering challenges. They include designing the lens, algorithm-driven sensors, and miniature electronic circuits that adjust the shape of the lens, plus creating a power source — all embedded within a soft, flexible material that fits over the eye.

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CHAMPAIGN, Ill. — A new class of miniature biological robots, or bio-bots, has seen the light — and is following where the light shines.

The bio-bots are powered by muscle cells that have been genetically engineered to respond to light, giving researchers control over the bots’ motion, a key step toward their use in applications for health, sensing and the environment. Led by Rashid Bashir, the University of Illinois head of bioengineering, the researchers published their results in the Proceedings of the National Academy of Sciences.

“Light is a noninvasive way to control these machines,” Bashir said. “It gives us flexibility in the design and the motion. The bottom line of what we are trying to accomplish is the forward design of biological systems, and we think the light control is an important step toward that.”

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