Hemophilia. Cystic fibrosis. Duchenne muscular dystrophy. Huntingtons disease. These are just a few of the thousands of disorders caused by mutations in the bodys DNA. Treating the root causes of these debilitating diseases has become possible only recently, thanks to the development of genome editing tools such as CRISPR, which can change DNA sequences in cells and tissues to correct fundamental errors at the sourcebut significant hurdles must be overcome before genome-editing treatments are ready for use in humans.

Enter the National Institutes of Health Common Funds Somatic Cell Genome Editing (SCGE) program, established in 2018 to help researchers develop and assess accurate, safe and effective genome editing therapies for use in the cells and tissues of the body (aka somatic cells) that are affected by each of these diseases.

Todaywith three ongoing grants totaling more than $6 million in research fundingDuke University is tied with Yale University, UC Berkeley and UC Davis for the most projects supported by the NIH SCGE Program.

In the 2019 SCGE awards cycle, Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering, and collaborators across Duke and North Carolina State University received two grants: the first will allow them to study how CRISPR genome editing affects engineered human muscle tissues, while the second project will develop new CRISPR tools to turn genes on and off rather than permanently alter the targeted DNA sequence. This work builds on a 2018 SCGE grant, led by Aravind Asokan, professor and director of gene therapy in the Department of Surgery, which focuses on using adeno-associated viruses to deliver gene editing tools to neuromuscular tissue.

There is an amazing team of engineers, scientists and clinicians at Duke and the broader Research Triangle coalescing around the challenges of studying and manipulating the human genome to treat diseasefrom delivery to modeling to building new tools, said Gersbach, who with his colleagues recently launched the Duke Center for Advanced Genomic Technologies (CAGT), a collaboration of the Pratt School of Engineering, Trinity College of Arts and Sciences, and School of Medicine. Were very excited to be at the center of those efforts and greatly appreciate the support of the NIH SCGE Program to realize this vision.

For their first grant, Gersbach will collaborate with fellow Duke biomedical engineering faculty Nenad Bursac and George Truskey to monitor how genome editing affects engineered human muscle tissue. Through their new project, the team will use human pluripotent stem cells to make human muscle tissues in the lab, specifically skeletal and cardiac muscle, which are often affected by genetic diseases. These systems will then serve as a more accurate model for monitoring the health of human tissues, on-target and off-target genome modifications, tissue regeneration, and possible immune responses during CRISPR-mediated genome editing.

Currently, most genetic testing occurs using animal models, but those dont always accurately replicate the human response to therapy, says Truskey, the Goodson Professor of Biomedical Engineering.

Bursac adds, We have a long history of engineering human cardiac and skeletal muscle tissues with the right cell types and physiology to model the response to gene editing systems like CRISPR. With these platforms, we hope to help predict how muscle will respond in a human trial.

Gersbach will work with Tim Reddy, a Duke associate professor of biostatistics and bioinformatics, and Rodolphe Barrangou, the Todd R. Klaenhammer Distinguished Professor in Probiotics Research at North Carolina State University, on the second grant. According to Gersbach, this has the potential to extend the impact of genome editing technologies to a greater diversity of diseases, as many common diseases, such as neurodegenerative and autoimmune conditions, result from too much or too little of certain genes rather than a single genetic mutation. This work builds on previous collaborations between Gersbach, Barrangou and Reddy developing both new CRISPR systems for gene regulation and to regulate the epigenome rather than permanently delete DNA sequences.

Aravind Asokan leads Dukes initial SCGE grant, which explores the the evolution of next generation of adeno-associated viruses (AAVs), which have emerged as a safe and effective system to deliver gene therapies to targeted cells, especially those involved in neuromuscular diseases like spinal muscular atrophy, Duchenne muscular dystrophy and other myopathies. However, delivery of genome editing tools to the stem cells of neuromuscular tissue is particularly challenging. This collaboration between Asokan and Gersbach builds on their previous work in using AAV and CRISPR to treat animal models of DMD.

We aim to correct mutations not just in the mature muscle cells, but also in the muscle stem cells that regenerate skeletal muscle tissue, explainsAsokan. This approach is critical to ensuring long-term stability of genome editing in muscle and ultimately we hope to establish a paradigm where our cross-cutting viral evolution approach can enable efficient editing in multiple organ systems.

Click through to learn more about the Duke Center for Advanced Genomic Technologies.

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Duke Researchers Garner Over $6 Million in NIH Funding to Fight Genetic Diseases - Duke Today

Venezuelan philosopher, Antonio Pasquali, who wrote extensively about how media and society affected each other, passed away on Oct. 5, 2019, in Spain.

In 1984, Pasquali was appointed Deputy Director General of the Communications Sector of UNESCO and Regional Coordinator for Latin America and the Caribbean of UNESCO from 1986-1989. He played an important role in UNESCOs New World Information and Communication discussions.

Pasqualis contributions to media studies are well-known in Latin America, but his research is less known in the English-speaking world. His research on media and communication inspired many Latin American scholars and media practitioners including myself who place ethics at the centre of the discussion.

Pasquali was a fierce critic of Canadian media theorist Marshall McLuhans view that the medium is the message that the medium in which things are disseminated determines their meaning. Always returning to human communication as the basis of relationships betwwen people, Pasquali warned us about the necessary conceptual and practical difference between communication and media.

For Pasquali, the ability to communicate is inherent to the formation of society. And so, any modification or control of communications becomes to a modification or control of society itself. He argued that technological changes, with their benefits and disruptions, have yet to transform the essence of human communication.

Pasqualis work is important to consider because he warned us about some troubling challenges that we can see around us.

Pasquali wrote about the ethics of communication, or what he called the moral dimension of communication. In his book 18 essays about communications, he identified six hard trends that would mark humanitys future:

1) A process of human-made environmental degradation that approaches the point of no return, as in the impending ecological crisis brought about by climate change and its consequences;

Read more: Dealing with the absurdity of human existence in the face of converging catastrophes

2) Human interference in natural evolutionary processes. He warned that advances in genetic engineering that bring hope for the treatment of diseases and also open the door to sophisticated mechanisms of social engineering and control;

3) Challenging the very idea of what being human is by: a) machines combined with living beings (cyborgs), and b) by the shift of human decision-making to artificial intelligence that could make humans irrelevant and even disposable. This will require new ways of understanding the relations between digital machines and human;

4) The persistence of nuclear, bacteriological, chemical and terrorist dangers, in a context of political polarization coupled with the emergence of extremist ideologies that could lead to internal and external violent confrontations;

5) The consolidation of the disparity between rich and poor that is already generating social unrest in different regions, as we have seen recently in Latin America and the Middle East;

6) The transformation of democracy into a plutocratic dictatorship (the government by the wealthy) based on the technological manipulation of social consensus, as illustrated in the Cambridge Analytica/Facebook scandal.

Pasquali was persistent in his struggle to establish a public broadcasting service in Latin American countries. His passion in defence of the need for a public media service never declined, and seems to be more relevant than ever in the midst of the Internet explosion.

Pasquali observed that the internet is now largely controlled by monopolies such as Facebook, Google, Amazon and Apple, and manipulated by big emerging powers like China. He vehemently denounced the communication hegemony of the authoritarian government of Hugo Chvez and his successor Nicols Maduro. Pasquali documented the setbacks that the regime has inflicted on Venezuelan society from the point of view of telecommunications, the media and transportation infrastructure.

At the end of his essay Will we communicate or inform ourselves?, Pasquali wondered if we are ready to give up a fundamental condition for our existence the ability and experience of communication. For him, communication was a mixture of intellect, passions and will that was intrinsic to how people and made meaning, personally and socially. He asked: Are we going to give up without a fight the possibility of communicating to another human being that we love him/her?

The great body of work that Pasquali produced will help us to answer these fundamental questions about the future of communication. Pasqualis intellectual legacy will live on through his writings and teachings.

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Society can be controlled through its means of communication - The Conversation CA

We are Earths Tech Support, declared Randall Kirk, Executive Chairman of the Board of Directorsand former CEO ofIntrexon. Intrexonis one of the biggest developers of synthetic biology (or engineering biology) applications in therapeutics, agriculture and chemicals. Kirk gave a keynote speech atSynbio Marketson synthetic biologys struggle to break into mainstream markets and its revolutionary new approach for industrial biotech in the food, pharmaceuticals, chemicals and materials sectors.

Before these new technologies can save the world, they need to be accepted and get to market. Companies must overcome the usual hurdles in finding investment and meeting regulatory requirements. They must find compatible scale-up partners and face new challenges in communicating the benefits and safety of their novel technology to society.

Partnerships for Success

Collaborations are beginning to blossom in synthetic biology. The field is often likened to the silicon chip industry. In its infancy, a single company would design, build and use their own chips. Now, companies outsource the design, building, testing and manufacture of chips along a structured value chain thanks to standardization of parts and uniformity in the field. This took years to achieve. Synthetic biology companies are currently developing their own unique tools to perform new feats in engineering biotechnology. Standardization is the dream and, to achieve this, companies must work together to break into the market.

A striking partnership at the conference was that ofAMSilkandAirbus. The airline industry has a problem: they must increase fuel efficiency by reducing weight of their aircraft without compromising on safety. Composite materials are an alternative to hefty sheet metals and AMSilk produces a durable but lightweight material: synthetic spider silk. AMSilk is interesting for its energy absorption, which is important for safety of the aircraft, Detlev Konigorski of Airbus explains. This partnership could help Airbus develop safe new materials while helping the carbon footprint of the airline industry.

One of the kings of collaboration isGinkgo Bioworks. Ginkgo uses several automated platforms to speed up and precisely carry out genetic manipulation, growth and testing of cells. To build their analytical power, they collaborated withBerkeley Lights, whose technology allows functional screening of thousands of cells simultaneously, increasing throughput.

Ginkgo has used this actively in their healthcare collaborations, such as a recent team-up withSynlogic, a microbiome therapeutics company developing living medicines. Ginkgo used its platform to increase the potency of SynlogicsE. coli-based drug in non-human primates in less than a year. Ginkgo CCO Matt McKnight wants to build on these partnerships by partnering with early-stage companies. Theyrecently announced a $350 m platformto build companies using Ginkgos foundries. He foresees more partnerships in the synthetic biology space in future, I think we shouldnt have full stack engineering biology companies. In any discipline, we dont see this. People work together.

Chemicals giantBASFis also interested in partnering with synthetic biology companies. Markus Pompejus, Vice President for Innovation and Scouting addressed the conference in Berlin citing the companys wide range of products. In principal, many products could be produced with biotech methods. Synbio is a research topic, but biotech is the application, Pompejus says.

Partnering may be off-putting for early-stage companies who want to maximize ownership of their company and the topic came up repeatedly at Synbio Markets. Where do you draw the line? Where do you co-develop with customers or should you do it more yourself? asks session chair James Hallinan ofCambridge Consultants, an expert engineering firm.

Depends where you are, says Alexandre Zanghellini of protein design companyArzeda, The later you partner, the more value you capture. You certainly want to keep the process propriety until the point where it can be scaled, then partner with marketing, scale up and development partners.

Talking Tech and Selling Solutions

Synthetic biology exists at the nexus of biology and nearly every other field. Its less a field of study and more of a precision engineering approach to traditional biotechnology using standardized tools and platforms. Kirk argued in his speech humanity has been using synthetic biology for thousands of years, using crop breeding as an example of humans precisely selecting and breeding desirable traits to engineer better strains of corn, for example. Now our role in the world has changed.

Weve been doing it for 12,000 years and weve been doing it without thinking of the consequences. Synthetic biology allows us tremendous specificity and potential to solve world problems by targeting individual species, he said.

How does this help us synthetic biology products access new and existing markets? Every process has biology in it, McKnight says.InscriptasCCO Jason Gammack thinks the solution lies in getting a few tangible products to lead the way. We need to make the products tangible. In the US were in hyperdrive mode. Two years ago, there was very little. Now,Impossible Foodsis in Burger King, says Gammack. Gary Lin ofPurple Orange Venturesthinks we need to raise the profile of synthetic biology among the public, adding One of the hard challenges, we need policymakers and government funding to support this. The amount of capital gone into this space is a drop in the ocean.

The issue spills over into the regulation of gene-edited technologies, especially in Europe. We recently had a debate on CRISPR plants, says Nadine Bongaerts-Duportet ofScience MattersandHello Tomorrow. The European Union regulations says CRISPR-edited crops are defined as genetically modified (GM), while those edited by radiation exposure are not. Bongaerts adds, The difference between UV exposure and CRISPR [as gene-editing methods], everybody understands the regulations dont make sense. How do you, with a positive message, make sure everyone gets it? All the panelists agree that building trust is key.

The trust us, were scientists approach doesnt work because people dont understand the technology, according to Gammack. I would fault all the synbio community, says Kirk. We look at polling data on GMO attitudes, I thought healthcare would be the first area [accepted]. In terms of polling, people have the greatest acceptance to insect disease vectors, he says, citing IntrexonsOxitecand theirGM mosquitoas an example.

The messaging, particularly around GM and especially here in Europe, is a minefield. From our perspective, we need to be mindful of potential roadblocks, says Lin, GM in food is the most difficult to grapple with. Part of the process is creating awareness of what the food process looks like. Transparency and openness about the technology is a major factor in getting this technology to market.MonsantosFlavr Savr tomatodisaster is still fresh in peoples minds. Public acceptance to this technology is a must before the market can be broken into reliably.

We need to understand emotions and backgrounds of people we talk to, to link our advancements to the incentives they care about. We should not over-hype, because if you can be critical and open about it, people will trust you, says Bongaerts.

Originally posted here:
Getting the Most from Biotech: Precision Engineering and Partnership - BioSpace

My hope is that we pay more attention to the way we talk about food; dietary regimes have become a means of self-definition a set of rules to follow, a clan to be a part of, which creates the potential to become divisive.

iStockPhoto / Getty Images

In this part of the world, it will be increasingly difficult for small agricultural operators to survive, let alone compete in the years to come, says Tony Marshall, who with his wife, Penny, runs a small-scale organic farm in High River, Alta., that has been in the family since 1899. He predicts that the increasing cost of land, equipment and labour could limit growth of small family farms, which are also increasingly impacted by fluctuating weather patterns. In addition, he foresees the cost and sophistication of regulatory compliance, food safety, organic certification, etc., will increase to the point of being prohibitive for smaller producers and processors, leaving only large enterprises able to afford the implementation and ongoing maintenance of complex programs such as the Safe Food for Canadians Act, which became law in January, 2019.

Certain segments of the public will continue to pursue connections with the people growing and making their food, but overall the marketplace will gradually slip away from any direct association to the land and their food sources, Marshall says. Highly processed foods like Beyond Meat and other lab-based alternatives will exacerbate this disconnect. Increasing food security demands from a growing global population will put even more pressure on agricultural producers to embrace technologies such as genetic engineering, farmed fish and factory-grown designer meat.

Feeding a hungry world is as dependent on government policies, distribution and food waste as it is upon increasing production outputs, Marshall says. Addressing the problem of food waste, on the farm and during transport, storage, processing, distribution, in retail and by end-users, will be essential to resolving food security issues.

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Im hoping restaurants will survive, restaurateur Tannis Ling says of the popular Bao Bei and Kissa Tanto in Vancouvers Chinatown, eateries that cultivate unique dining experiences with attention to decor, music, service and other details that are lost when food is ordered via delivery options, such as Uber Eats and DoorDash. Ling sees an increasing desire for fast casual, takeout and non-committal eateries that focus on just a few items, such as Juke Fried Chicken across the street from Bao Bei. Those who are doing it well are killing it, she says.

Though she has witnessed a dramatic shift in restaurant culture over the decades cooks, kitchen and front of house staff are paying more attention to their physical and mental health, and prioritizing a more manageable work-life balance Ling worries, as does the back of house operations manager Alain Chow, about staffing becoming even more challenging in the coming years. In a gig economy, many young people who have traditionally sought jobs in the food industry are opting to make money doing multiple smaller tasks, many of them virtual or digital. Were still finding passionate young cooks, Chow says, but retaining them is the hard part.

They both expect technology to have an ever-increasing presence in the coming decade, citing a local hot pot restaurant at which diners order from their tables on iPads, but believe that social media has hit a plateau in terms of its influence on the dining experience. With such a constant barrage of imagery and information, Ling says, it no longer has the same impact.

Big changes are gradual, almost imperceptible, but were in the midst of one, says educator, activist and historian Anita Stewart, who has been writing about food in Canada for almost 40 years, and this year will see the creation of her namesake Alumni Food Laboratory at the University of Guelph, a multifunctional lab and teaching studio that will enable hospitality and nutrition students to learn innovative practices in food preparation, production and food science.

Our food supply is among the best in the world and Canadian chefs, restaurateurs and even grocery chains with branding programs have adopted local/regional foods as a point of differentiation, she says. Even a generation ago, locally grown food was not commonplace on restaurant menus; today its almost a given that restaurants will support local farms and producers. Stewart sees that trend continuing, propelled by a rise in concern over the impact our food choices have on the environment. Reducing the miles our ingredients travel has to be a factor, she says.

My hope for the coming decade is that the industry be more collaborative, Stewart says. Look down, on whose shoulders are we standing? Look beside, whos travelling on a similar pathway? Look ahead, there are way too many silos and we need to build some bridges. We all, as consumers, need to be more grateful, introspective and well-informed advocates for Canadian agricultural and processing communities who are competing not only with the world, but increasingly devastating weather.

I probably check the weather three times a day, says Ann Sperling, winemaker and director at Canadas first biodynamic vineyard and winery, Southbrook Vineyards, in Ontarios Niagara on the Lake, and owner and winemaker at Sperling Vineyards in Kelowna, B.C.

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Covering two growing regions in Canada, she says our wine industry is flourishing, and yet all regions Nova Scotia, Quebec, Ontario and British Columbia have had challenges in recent years, much related to erratic weather. More investments are being made to develop technology to mitigate environmental instability issues; textiles to cover vines in colder regions, wind machines to minimize frost risk and irrigation for periods of drought are just some of those innovations. But there are still no systems in place to deal with crop failures, and government has been reducing its support for the industry, and agriculture in general. Theres no bigger picture safeguard, she says. Were more on our own.

The more people realize there is an impact on climate, that C02 levels are rising, the more people will start to make their buying decisions based on environmental impact, Sperling says. The good news: By next year, B.C. will have one of the highest percentages of organic vine production in the world, at around 12 to 15 per cent.

Because, generally, we do not find wine as intimidating as it used to be, Sperling doesnt see new cannabis legislation affecting the industry; the two fit a different niche in peoples lives. And much of the industrys marketing is geared toward younger consumers, who have increasing buying power without the same biases of generations past. Theyre more inclined to try new things, Sperling says, and they think more globally in terms of how they spend their money.

A shift in attitude toward food excites me. At home, people are paying attention to food waste and learning to cook more intuitively, approaching mealtimes from the standpoint of whats in the fridge that needs to be used, rather than sourcing specific ingredients for a recipe they perhaps saw online. Home cooking is becoming more valued and less competitive. People are sharing more meals at home, and even at work. My hope is that we pay more attention to the way we talk about food; dietary regimes have become a means of self-definition a set of rules to follow, a clan to be a part of, which creates the potential to become divisive.

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How we'll eat in the decade ahead - The Globe and Mail

Jack Duffy-Protentis said he has enjoyed working with his hands since childhood and looks forward to working in the automotive industry.

EASTON Jack Duffy-Protentis is less than a year away from graduating and starting a career as an engineer, a field that he enjoys because it allows him to work with his hands.

In addition to being a college student and learning concepts from chemistry and physics he has applied to his work, the Easton native has faced another challenge: being visually impaired.

"I do the same thing as everyone else, but I just have to find a different path," said Duffy-Protentis, who attends Worcester Polytechnic Institute and studies mechanical engineering.

Being visually impaired is challenging, the 22-year-old said. It adds more steps and time to do things, but that isn't a barrier.

"It's not stopped me from doing anything other than driving," Duffy-Protentis said.

His family realized that he had difficulty seeing in kindergarten when he struggled to read and write.

In the third grade, Duffy-Protentis was diagnosed with Stargardt's disease, a rare degenerative genetic disease that affects the retinas.

Duffy-Protentis recalled feeling relieved that he wasn't stupid. For his parents, the diagnosis was more difficult and they wanted to find a way to help him, whether it was through treatment or support.

"Part of Jack's success now and in life is that he doesn't have an attitude that is defeated," said his father Paul. "He's optimistic. He doesn't look at obstacles as obstacles. There are just challenges to overcome."

Today, Duffy-Protentis has about 20 percent of his vision left. His peripheral vision is good, but his central vision, where most details come from, has decreased.

He can make out shapes. When Duffy-Protentis looks at people, he looks at the tops of their heads and can understand where their features are, but can't see their facial expressions.

He has been interested in tinkering with things and later found that engineering would be a good fit for him. Duffy-Protentis wanted to find a school with a hands-on approach to learning like WPI.

After graduation in December, he wants to go into automotive engineering and work abroad with hypercar companies like Lamborghini, Ferrari or Sweden-based manufacturer Koenigsegg.

"Everything is mechanical and working in sync and relating to another system," Duffy-Protentis said about his interest in cars. "It all sort of comes together."

Before college, he restored a 1964 Mustang. Despite his diminishing vision, Duffy-Protentis has continued to work on it by using his sense of touch.

There were also other signs that he was mechanically inclined. Duffy-Protentis liked building toys, like Legos, and was a co-captain of the Oliver Ames High School robotics team.

He is also the only science person among his three siblings and cousins.

WPI is academically challenged and fast-paced, he said. For his senior project , Duffy-Protentis is working with a team of students to convert a gasoline jet ski into a solar energy and electric one. The goal is to build a prototype.

In class, he sits at the front of the room and takes pictures of notes on the board. He meets daily with a tutor to make sure he understood material from lectures.

To navigate, he has a guide dog named Adonis. Duffy-Protentis and the 2-year-old yellow Labrador retriever have been together since May after they graduated from a training school called Guiding Eyes for the Blind.

Duffy-Protentis said he is the first guide dog user at his university.

When he's not on the job, Adonis is a regular dog who likes to play and receive. Once the harness is on, he's focused.

Having Adonis has helped Duffy-Protentis feel more approachable, he said. When using a cane, people didn't really know how to act around him, but with a dog, he said people are more likely to interact with him or ask questions.

Adonis, the guide dog, has kept him company, but Duffy-Protentis has also made an effort to combat the social isolation of blindness.

His solution has been to become more of an extrovert and to introduce himself to people around campus.

His other interests include outdoor activities, like hiking, and board sports, like skiing and surfing. Being visually impaired helps him balance better, he said.

Duffy-Protentis is a member of the Society of Automotive Engineers and the Phi Sigma Kappa fraternity.

He knew that his fraternity was a good match for him when his brothers made an effort to include him in one of its traditions.

After members are accepted into the fraternity, they play a game of football. Duffy-Protentis planned to sit the game out because he can't see the ball when it's in the air.

Fraternity members asked if he could play soccer, which Duffy-Protentis can because he can see it better. The fraternity altered the tradition so that he could participate.

Beyond school, Duffy-Protentis has interned at a manufacturing center in Easton called Case Assembly Solutions that makes circuit boards.

He has volunteered a science, technology, engineering and math camp for children through the National Federation for the Blind and likes that he can be an example of a visually impaired engineer.

"There aren't many visually impaired engineers out there," Duffy-Protentis said.

Staff writer Mina Corpuz can be reached at mcorpuz@enterprisenews.com. Follow her on Twitter @mlcorpuz.

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Visually impaired Easton native finds a different path as an engineer - Enterprise News