Category Archives: Human Genetic Engineering

Paul Vincelli, extension professor and Provosts Distinguished Service Professor at the University of Kentucky| March 7, 2017

HIGHLIGHTS:

Biases, conflicts of interest come from many sources, including associations with industry, advocacy groups, other non-profits Industry funding of studies on GE crops does not appear to be important bias source Personal experience suggests corporations receptive to negative results, as they improve products, limit liability Limited resources for much agricultural research without industry support Dubious shill accusations against biotech scientists discourage public engagement, depress discourse

Agricultural scientists who interact with the public often feel under enormous scrutiny. One of the most common concerns is that professional ties with industryespecially obtaining funding from industrycompromise scientific credibility. This concern is particularly acute in the area of genetically engineered crops (GE crops, commonly known as GMOs).

Research into genetically engineered crops is not my specialtymy work is focused on plant pathologyand I have never solicited nor received private-sector funding on this issue. Over my career, my industry interactions have dealt with non-GMO products for plant disease control. My interest in GE crops arises from their potential to address genuine human needs and to reduce the environmental footprint of agriculture. And I am concerned that a dark shadow has been cast over many independent scientists because of their collaborative efforts with various stakeholders, including companies.

Biases From Many Sources

Across multiple disciplines, industry-funded projects may be more likely to report positive outcomes, or less likely to report negative outcomes [1-4]. However, industry funding is not always associated with biased outcomes [5, 6]. Furthermore, many sources of funding NGOs, non-profits, other civil and governmental organizationsmay engender conflicts of interest (COIs) and biases that influence reported research. Powerful biases may arise for non-monetary reasons [7] in both researchers and in non-researcherspossibly including you and me.

Regarding GE crops, I am aware of three journal articles on the topic of industry funding and bias. In the first [8], the authors found no evidence of bias due to financial COIs (studies sponsored by an industry source that may benefit from the outcome), but they did document bias associated with professional COIs (where at least one author was affiliated with a company that could benefit from the study outcome). In that study, among the 70 studies examined (see their Table 2), 61% had either a financial or a professional COI. Among the much larger sample size (698 studies) examined by Sanchez [9], the majority had no COI, and only one quarter had COIs related to author affiliation and/or declared funding source.

A recent study by Guillemaud et al [10] had similar findings: among 579 studies with definitive COI information (see their Figure 3), the majority did not report a COI. However, among those with COIs, there was a higher probability of reported outcomes favorable to the GE crop industry. In addition to these journal articles, another independent analysis [11] suggested that industry funding did not bias study outcomes for GE crops, but these data have not been analyzed statistically nor published in a peer-reviewed journal.

Thus, while evidence to date shows that the majority of studies on GE crops are not influenced by COIs, some fraction is so influenced. Therefore, there is value in remaining alert to the possibility of bias and in continuing to practice full disclosure. I believe it is important to remain alert to COIs and biases of all sortsnot only those associated with corporate influences, but also those of NGOs or other civil organizations that may have explicit or implicit agendas.

Some people simply do not trust corporations. This is understandable, given the indefensible behavior of some in business, such as the tobacco industry, the chemical industry, Exxon, and Volkswagen [12-15]. Consequently, some members of the public perfunctorily dismiss commercial-sector scientists who may have solid scientific skills and high personal integrity. I personally must admit to a measure of distrust of corporations, which may even express itself occasionally as an anti-industry bias. But I also believe it is unwise to categorically reject all industry-funded data, solely on the basis of their provenance. In fact, I would label such an attitude a bias itself. Thoughtful, evidence-based analysis must always trump bias and ideologyand does, for a good scientist.

Why do researchers accept industry funding? Public-sector and private-sector scientists may share common interests. Industry scientists and I share a common interest in knowing what works in the field and what doesnt. Consequently, industry sources provide funding for field tests of their products for plant disease control. Furthermore, public funding for science in the USA is insufficient to support even a fraction of the worthy research projects. Inadequate funding can quickly and thoroughly undercut a career in science at any stage. Since researchers are hired to do research on important topics and not to whine about the difficult state of public funding, some will welcome funding from commercial sources, if it allows them to continue to do research they believe is intellectually compelling, important to society, or both. Also, industry scientists may have knowledge, skills, and facilities that we public scientists may not.

My Funding Choices: Scientific Rigor Coupled With Personal Integrity

Discussing my own practices should provide an idea of how many scientists work. Roughly half of my funding over the years has been from industry, primarily to support product testing for plant disease control. I have commonly tested synthetic fungicides, but I have also tested natural products of various sorts. In fact, commercial pesticide manufacturers can fairly accuse me of an anti-pesticide bias. I say this because I have tended to favor testing products that might be perceived as more consistent with sustainability (biocontrol products, for example) than applications of synthetic chemicals, often requesting limited, or no, funding for such tests. Besides industry funding, I have received federal funds for research and outreach on detection and management of plant diseases.

I publish all efficacy trials in Plant Disease Management Reports. We commonly publish data showing inadequate efficacy or phytotoxicity, and I never consider funding sources when the report is drafted. In fact, the reports are drafted by the Senior Research Analyst who conducts the field work, and he doesnt know who provided funding nor for what amount. Thus, our testing program does not suffer from publication bias. This approach is not exceptional [16, 17].

I accept no personal giftsmonetary or materialfrom private-sector sources.

I have no hesitation about challenging multinational corporations. For example, I provided a degree of national leadership in challenging a major pesticide manufacturer over certain uses of a commercial crop fungicide. I was one of the lead authors of a letter to the US Environmental Protection Agency raising questions about the paucity of public data to support plant health claims. I gave a similar talk in a major scientific conference, the 2009 American Phytopathological Society meeting.

Several factors may help me and other scientists to offset natural human tendencies towards bias:

A common concern is that providing funding buys access to researchers. This may sometimes be the case, but for me, this criticism doesnt fit. I am an Extension Specialist everybody has access to me and my expertise. I dont recall a single instance in my entire career when I failed to return a phone call or email from anyone. In fact, it is a federal requirement that Extension programming be grounded in engagement with diverse stake- holdersincluding, but certainly not limited to, industry [18].

What Happens When Data Fall Short Of Company Expectations?

We regularly see poor product performance in our experiments. In a memorable instance, we observed visible injury to a creeping bentgrass putting green from a particular formulation of the widely used fungicide, chlorothalonil. On the day of application, the turfgrass was suffering exceptionally severe drought stress, due to an irrigation equipment failure, which probably was a predisposing factor.

I notified the company of my observations, which is my standard practice if a product provides unexpectedly poor performance or unexpected phytotoxicity. This is not to provide the company the opportunity to help me see the error of my ways. Rather, this is simply good scientific practice. I want industry scientists to collect their own samples, so that they may better understand the poor results obtained; and to offer hypotheses or insights that may account for the unexpected results, as they often know things about their product and its performance that I do not.

In the case of the turfgrass injury caused by chlorothalonil, a company representative and I visited the experiment together and shared observations. I listened to the representatives hypotheses and shared my own. After the meeting and additional lab work, I reported my findings in various outlets. In my research program, unfavorable results get reported no differently than favorable results.

I must state emphatically that, in my 34 years of product testing for plant disease control, I cannot recall a single instance where a company representative attempted to pressure me to report favorable results. Company representatives do not like to receive bad news, but in my experience, almost every company representative I have interacted with has been professional enough to recognize the importance of discovering the limitations of their products sooner rather than later. The consequences of introducing an inadequate product can be catastrophic for a corporation.

Corporate Funding for Outreach

What about private-sector funding for outreach? To my knowledge, such funds are never provided with a quid pro quo that the scientist will make particular claims about a companys products. To the contrary, private-sector representatives take note of speakers whose scientific understanding is consistent with their own. They may approach those speakers to discuss possible support for outreach, but without specifying the content of such presentations. Although I refuse industry funding for all aspects of GE crops, I do not suspect undue industry influence when funds are provided for travel expenses or supplies of invited speakers. Even honoraria or stipends for speaking engagements dont particularly concern me. This is true for such funding across the full spectrum of possible funding sources, ranging from advocacy groups for organic agriculture to multinational pesticide manufacturers. I want to see the scientific methods and data, no matter who did the study.

Who Should Pay For Research?

Should publicly funded professors even do product testing? Yes: there is a public interest in independent assessments of how products perform. The more public data on performance, the better.

If you agree that third-party testing is desirable, the question arises, Who pays for it? I believe that, usually, the manufacturer is responsible, not the taxpayer. Of course, this raises concern about funding bias. If a researcher wishes to avoid funding bias, can they tap into other sources? Not in my discipline. Pools of public funding for product testing are essentially non-existent.

What about studies of possible impacts of products to the environment? Who should pay for that? Again, in my opinion, such costs fall to the manufacturer, although in some cases, there is a compelling public interest that justifies the use of public funds for product testing.

Final thoughts: Does industry-researcher cooperation undermine the credibility of scientific research?

For me, the answer is, No. We should be cognizant of possible biases and COIs due to source of fundingwhether the source is industry, NGOs, advocacy organizations, or other sources. Disclosure is critical [7, 19]. However, industry scientists are often excellent scientists who take pride in their work, no differently than any industry critic. Yes, we should exercise a degree of caution when reviewing industry-funded research, but the same holds for research funded by advocacy organizations, since each has an agenda. Personally, in all cases, I will not reject either source out of hand; I will judge the work based on its scientific merit.

Sometimes the bias against industry-funded research on GE becomes hurtful, especially in the social media. Witnessing dedicated public servants being unfairly attacked as industry shills is demoralizing to public scientists, and it has the unintended consequence of discouraging public engagement by scientists who already have very busy professional and personal lives. Such unfounded charges are not only divisive and unproductive: they are unkind and can be abusive. (Sadly, unkind behavior can be found in all sides of the GMO debate.)

My freedom from industry funding on all aspects of GE protects me from similar accusations. Yet it doesnt surprise good scientists that, after years of studying the scientific literature, I independently arrived at an understanding very similar to that presented in the re- port of the National Academy of Sciences, Engineering and Medicine (NASEM) published earlier this year [20]. This isnt because industry has somehow influenced me or the members of the NASEM review committee. It is because there is a substantial body of credible science supporting the conclusions presented in the NASEM report. In reviewing the body of peer-reviewed scientific literature on GE crops, one is likely to arrive at similar conclusions. I had an identical experience with the scientific consensus on climate change [21].

Ultimately, with enough careful study of evidence from credible sources, fidelity to good scientific practice, and a degree of humility, it is hard not to arrive at findings rather similar to those of journal-published experts of a scientific discipline. They actually do know something about their subject after all.

Paul Vincelli is an Extension Professor and Provosts Distinguished Service Professor at the University of Kentucky. Over the 26 at UK, he has developed specializations in management of diseases of corn, forages, and turfgrasses, molecular diagnostics, and international agriculture. He also has provided Extension programming on climate change and on genetic engineering of crops. He currently is UKs Coordinator for the USDAs Sustainable Agriculture Research and Education program, and he serves as Councilor-At-Large for the American Phytopathological Society.

The Genetic Literacy Project is a 501(c)(3) non profit dedicated to helping the public, journalists, policy makers and scientists better communicate the advances and ethical and technological challenges ushered in by the biotechnology and genetics revolution, addressing both human genetics and food and farming. We are one of two websites overseen by the Science Literacy Project; our sister site, the Epigenetics Literacy Project, addresses the challenges surrounding emerging data-rich technologies.

Acknowledgements

Thanks are expressed to John R. Hartman and Jon Entine, for reviewing earlier drafts of the manuscript.

Disclosure Statement

The author declares no conflicts of interest in the topic of GE crops. Detailed disclosure documents may be found here. The author donated the full amount of his monetary honorarium for writing this article to Human Rights Watch.

Literature Cited

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8. Diels, J., Cunha, M., Manaia, C., Sabugosa-Madeira, B. and Silva, M., Association of financial or professional conflict of interest to research outcomes on health risks or nutritional assessment studies of genetically modified products. Food Policy, 2011, Vol. 36, p. 197-203, DOI: 10.1016/j.foodpol.2010.11.016. Available from: http://www.sciencedirect.com/science/article/pii/S0306919210001302

9. Sanchez, M. A., Conflict of interests and evidence base for GM crops food/feed safety research. Nat Biotechnol, 2015, Vol. 33, p. 135-7, DOI: 10.1038/nbt.3133. Available from:http://www.ncbi.nlm.nih.gov/pubmed/25658276

10. Guillemaud, T., Lombaert, E. and Bourguet, D., Conflicts of interest in GM Bt crop efficacy and durability studies. PLoS One, 2016, Vol. 11, p. e0167777, DOI: 10.1371/journal.pone.0167777. Available from:https://www.ncbi.nlm.nih.gov/pubmed/27977705

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12. Gates, G., Ewing, J., Russell, K. and Watkins, D. Explaining Volkswagens Emissions Scandal. New York Times. 19 Jul 2016. Available from: http://www.nytimes.com/interactive/2015/business/international/vw-diesel-emissions-scandal-explained.html.

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Should University Agricultural Research Scientists Partner With Industry? - Genetic Literacy Project

Facility to Provide Clinical Supply of Lead Gene Therapy Programs TN-201 and TN-401 for Planned First-in-Human Studies

94,000 sq. ft. Modular Facility has Initial Production Capacity at the 1000L Scale

SOUTH SAN FRANCISCO, Calif., June 16, 2022--(BUSINESS WIRE)--Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a biotechnology company with a mission to discover, develop and deliver curative therapies that address the underlying causes of heart disease, today announced that it has completed the build-out and operational launch of its Genetic Medicines Manufacturing Center in Union City, California. Tenaya is advancing a pipeline of therapeutic candidates, including several adeno-associated virus (AAV) gene therapies, for the potential treatment of both rare and prevalent forms of heart disease.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220616005336/en/

Tenayas Genetic Medicines Manufacturing Center located in Union City, CA (Photo: Business Wire)

"Tenaya made an early, strategic commitment to internalize several core capabilities to optimize the safety, efficacy, and supply of our product candidates on behalf of patients. With todays announcement we have made a big leap forward on that commitment by establishing end-to-end in-house manufacturing capabilities for our pipeline of AAV-based gene therapies," said Faraz Ali, Chief Executive Officer of Tenaya. "The operational launch of Tenayas Genetic Medicines Manufacturing Center represents an important milestone as we prepare to advance our robust pipeline of potentially first-in-class cardiovascular therapies into initial clinical studies."

Tenayas Genetic Medicines Manufacturing Center is designed to meet regulatory requirements for production of AAV gene therapies from discovery through commercialization under Current Good Manufacturing Practice (cGMP) standards. Initial production efforts will support first-in-human studies of Tenayas lead gene therapy, TN-201. TN-201 is being developed for the treatment of genetic hypertrophic cardiomyopathy (HCM) due to MYBPC3 gene mutations. Tenaya plans to submit an Investigational New Drug (IND) application for TN-201 to the U.S. Food and Drug Administration (FDA) in the second half of this year. The facility will also support cGMP production for TN-401, Tenayas gene therapy program being developed for the treatment of genetic arrhythmogenic right ventricular cardiomyopathy (ARVC) due to PKP2 gene mutations, for which the company plans to submit an IND to the FDA in 2023.

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"The investment in our own world-class manufacturing facility provides Tenaya with greater control over product attributes, quality, production timelines and costs, which we believe will ultimately translate into better treatments for patients," said Kee-Hong Kim, Ph.D., Chief Technology Officer of Tenaya Therapeutics. "Tenayas Genetic Medicines Manufacturing Center complements our established internal genetic engineering and drug discovery capabilities and is designed to meet our near- and long-term needs such that we can readily scale and expand as our pipeline matures and evolves."

Tenaya completed customization of approximately half of the 94,000 square foot facility to incorporate manufacturing suites and labs, office space and storage. Utilizing a modular design, the state-of-the-art facility is now fully operational with initial capacity to produce AAV-based gene therapies at the 1000L scale, utilizing Tenayas proprietary baculovirus-based production platform and suspension Sf9 cell culture system. The excess space and modular design of the Genetic Medicines Manufacturing Center is intended to provide Tenaya with considerable flexibility to expand manufacturing capacity by increasing both the number and the scale of bioreactors to meet future clinical and commercial production needs.

The Union City location, approximately 30 miles from Tenayas South San Francisco headquarters, is expected to enable the seamless transition of Tenayas science from early research through commercial manufacturing. The selection of this location is intended to foster a culture of close collaboration across teams at all stages of developing and testing novel AAV capsids, de-risk the translation from research to process development and create opportunities for improvements in production processes. The Genetic Medicines Manufacturing Center is staffed by a growing in-house team with expertise in all aspects of gene therapy manufacture, including process development, analytical development, quality assurance and quality control.

About Tenaya Therapeutics

Tenaya Therapeutics is a biotechnology company committed to a bold mission: to discover, develop and deliver curative therapies that address the underlying drivers of heart disease. Founded by leading cardiovascular scientists from Gladstone Institutes and the University of Texas Southwestern Medical Center, Tenaya is developing therapies for rare genetic cardiovascular disorders, as well as for more prevalent heart conditions, through three distinct but interrelated product platforms: Gene Therapy, Cellular Regeneration and Precision Medicine. For more information, visit http://www.tenayatherapeutics.com.

Forward Looking Statements

This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as "potential," "will," "plans," "believe," "expected," and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, statements regarding the therapeutic potential of Tenayas pipeline of therapeutic candidates; Tenayas plan to use the cGMP manufacturing facility for the production of TN-201 and TN-401; Tenayas belief that its cGMP manufacturing facility will enable seamless transition from early research through commercial manufacturing and translate into better treatments for patients; the expected timing for submission of IND applications for TN-201 and TN-401; and statements by Tenayas chief executive officer and chief technology officer. The forward-looking statements contained herein are based upon Tenayas current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early stage company; Tenayas ability to successfully manufacture product candidates in a timely and sufficient manner that is compliant with regulatory requirements; Tenayas ability to develop, initiate or complete preclinical studies and clinical trials, and obtain approvals, for any of its product candidates; the timing, progress and results of preclinical studies for TN-201, TN-401 and Tenayas other programs; Tenayas ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Tenayas manufacturing and operations, including preclinical studies and planned clinical trials; the timing, scope and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; Tenayas manufacturing, commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenayas reliance on third parties; Tenayas ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled "Risk Factors" in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220616005336/en/

Contacts

Investors Michelle CorralVice President, Investor Relationship and Corporate CommunicationsTenaya TherapeuticsIR@tenayathera.com

Media Wendy RyanTen Bridge CommunicationsWendy@tenbridgecommunications.com

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Tenaya Therapeutics Launches Operations of New Genetic Medicines Manufacturing Center to Support the Development of Potentially First-In-Class...

Albany NY, United States: CRISPR cas systems are commonly used in microbial engineering that includes immunization of cultures, bacterial strain typing, and self-targeted cell killing. Further, CRISPR and cas genes market system is also applied to control metabolic pathways for an improved biochemical synthesis. This technology is also used for the improvement of crop production. These factors further drive growth in the CRISPR and cas genes market.

CRISPR and cas genes system has been a revolutionary initiative in the biomedical research field. The application of this technology in somatic cell genome editing events has targeted to its application. The technologies are commonly used for the treatment of different genetic disorders. But, the ethical issues while using the system from the CRISPR and cas genes market are somewhere curtailing the growth in the industry. Furthermore, the market is also witnessing a lack of proficient professionals, which restrains its growth opportunities.

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The market forecast on CRISPR and cas genes market was estimated US$ 1,451.6 Mn. Now it is predicted to climb US$ 7,234.5 Mn during forecast period from 2018 to 2026. The market is estimated to reach a compound annual growth rate (CAGR) of 20.1% from 2018 to 2026.

Multiple Applications and Diverse Dominating Factors in CRISPR and Cas Genes Market

The report from market research on CRISPR and cas genes industry has marked its division on the basis of region, end-user, application, and product type. DNA-free cas and vector-based cas are the two types in which the CRISPR and cas genes market is bifurcated on the basis of product type. Between these two types, the vector-based cas section has dominated the market at international levelin 2017. This expression system is helpful for the researchers who are focusing to enrich Cas9-expressing cells and concentrate on the establishment of a stable cell line. The vector-based cas is available with an analytical that is used to support the creation of durable cell lines. These lines are designed with minimal possible background expression.

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The major advantages of the DNA-free cas segment boost growth in the CRISPR and cas genes market. DNA-free cas components are used for the reduction of potential off-targets. They also find application to trace correlations with human illnesses.

Knockout/activation, functional genomics, disease models, and genome engineering are the classification types in the CRISPR and cas genes market on the basis of application in different verticals. Contract research organizations, government and academic research institutes, pharmaceutical and biotechnology companies are some of the key end-use industries in the market. Further, as per the market analysis report on CRISPR and cas genes market, the industry is spread in different regions that include Middle East & Africa, Latin America, Asia Pacific, Europe, and North America.

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The industry players from market have adopted inorganic and organic growth strategies for the expansion of product offerings, capturing market share, increasing consumer base, and strengthening geographical reach. Some of the key players in the CRISPR and Cas genes market include Dharmacon, Synthego, GenScript, OriGene Technologies, Inc., Applied StemCell, Inc., Addgene, and Cellecta, Inc.

Genome Engineering to Dominate CRISPR and Cas genes market

On the basis of application, the genome engineering section has dominated in the CRISPR and cas genes market. The genetic materials can be added, detached, and altered with the help of CRISPR technology at any specific location in the genome. Genomic engineering is related to the synthetic assembly of comprehensive chromosomal DNA, and it has been commonly taken from natural genomic sequences.

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The CRISPR and Cas genes market has been dominated by pharmaceutical and biotechnology companies in terms of end-user. The strategic partnerships and innovations may boost growth in the market.

North America and Europe are the regions that account for the maximum share in the CRISPR and Cas genes market. Rising technological advancements and research activities are driving growth in the market.

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CRISPR and Cas Genes Market is Anticipated to Reach US$ 7,234.5 Mn by 2026, Increase in Incidence of Genetic Disorders to Drive the Market - BioSpace

BySydney Portale

Vaccines are a critical tool in the protection of humans and animals against pathogens, but a major challenge for vaccine development is understanding why vaccines work better for some individuals than others.

To answer this question, a research team led by Yana Safonova, assistant professor in the Department of Computer Science at the Johns Hopkins Whiting School of Engineering, studied black angus cows and their varying responses to the Bovine Respiratory Disease, or BRD, vaccine. The team's findings were recently published in the journal Genome Research.

BRD is the leading cause of natural death for cows and costs the cattle industry an estimated $900 million a year. Medication is expensive, so cattle producers rely on vaccinations to mitigate the problem.

Conducting research for the U.S. Department of Agriculture, Safonova and researchers from the University of California, San Diego sought to understand how the unique genetic structure of cows and other bovine animals such as bison, buffalo, and antelopes were creating antibodies from the BRD vaccine.

A large-scale study of human immunogenetics could aid in understanding vaccine response variations ahead of the next pandemic.

"We wanted to answer one particular question: Why are some individuals within the population of black angus cows responding very differently to the same vaccine?" Safonova said.

The researchers examined a distinguishing feature of bovine immunity: the long complementarity-determining region H3 loops in the antibodies they create. Bovine antibodies with such ultralong CDR H3 loops have been found to neutralize certain strains of HIV, and Safonova and her team have discovered that they are also one key to developing antibody responses against BRD.

Using a new computational tool that they designed, Safonova and her team analyzed sequencing data from antibodies produced by the black angus cow population and pinpointed genetic variations in antibodies associated with immune responses.

The researchers found that while the creation of these unique antibody structures was triggered by each vaccine dose, vaccine efficacy (how well the vaccine actually works) is determined long before the individual mounts an immune response. Segments of DNA called variable, diversity, and joining immunoglobulin genes, also referred to as IG genes, are what produce antibodies and control individual responses to a vaccine.

This means vaccine efficacy for an individual is pre-determined before the vaccine is even administered.

Because the team's method can reveal these genetic markers, cattle producers could potentially use this information to selectively breed cows that are less susceptible to BRD based off their genetic predisposition, said Safonova.

The researchers say that their study is the largest personalized immunogenetics study across any species to date, and that their results open doors to applying immunosequencing to human vaccine studies. In-depth immunogenetics research would allow scientists to discover patterns in the human genome that determine the body's programmed response to vaccines. In fact, Safonova says a large-scale study of human immunogenetics could aid in understanding vaccine response variations ahead of the next pandemic.

Safonova explained, "With new strains of COVID-19, new variants, and the need for vaccinations, we can show that this type of study will work for many different subjects. We want to highlight how we can study [the vaccination process] across different genomes."

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Large-scale bovine vaccine study reveals the role of genetics in immune response - The Hub at Johns Hopkins

Gene editing, which allows precise edits to the genome, has been widely used for a variety of applications in laboratories worldwide since its discovery a decade ago. It has tremendous potential: Researchers hope to use it to alter human genes to eliminate diseases; improve the characteristics of plants; resist pathogens; and more. The two scientists who discovered the iconic gene editing technology, the CRISPR-Cas9 system, were awarded the 2020 Nobel Prize in Chemistry.

In spite of the fact that gene editing is essentially a refinement of earlier, less precise, less predictable techniques for genetic modification, finding the right approach to regulating it has been elusive. Initially, many nations treated it as a stringently regulated GMO, or genetically modified organism, which posed conceptual problems from the outset. For one thing, theres really no such thing as a GMO, except in the fevered imagination of bureaucrats, legislators, and activists, but that didnt prevent this pseudo-category from being subjected to onerous regulation.

Genetic engineering, or genetic modification, is a seamless continuum of techniques that have been used over millennia, including (among others) hybridization, mutagenesis, somaclonal variation, wide-cross hybridization (movement of genes across natural breeding barriers), recombinant DNA, and nowgene-editing. The primary distinction between the last two and the others is they are far more precise and predictable than the earlier techniques.

Since the advent in the 1970s of recombinant DNA technology, which enables segments of DNA to be moved readily and more precisely from one organism to another, molecular genetic engineering techniques have become ever more sophisticated, precise, and predictable. This evolution has now culminated in the most recent discoveries, the CRISPR-Cas9 system and variations of it. Its a way to find a specific bit ofDNAinside a cell and then to alter that piece of DNA. CRISPR can also be used to turn genes on or off without altering their sequence.

CRISPR(short for Clustered Regularly Interspaced Short Palindromic Repeats) is a natural defense system that a range of bacteria use against invading viruses. CRISPR can recognize and guide the system to specific DNA sequences, while the enzyme Cas9 (or other Cas proteins) cuts the DNA at the recognized sequence. As often happens in science and reminiscent of mutagenesis a century ago and recombinant DNA technology in the 1970s molecular biologists and genetic engineers quickly copied and adapted the naturally occurring system. Using CRISPR-Cas9, scientists can target and edit DNA at precise locations, deleting, inserting or modifying genes in microorganisms, plants and animals, including humans. CRISPR-Cas9 is cheaper, faster, easier, more precise, and more predictable than its genetic engineering predecessors, and scientists are continuously improving the technique, its predictability and safety.

The USand Canada have deregulated gene-edited organisms in principle, moving towards risk-based regulation, while Europe, with its long-standing, intractable opposition to genetic engineering, has decided to equate gene editing with heavily regulated, and sometimes even banned, GMOs. Other nations, such as the UK, are beginning to move away from Europes hostile regulatory climate, with hopes of improving their farmers livelihoods as well as finding new partners for international food trade, besides their European friends.

The polar extremes of acceptance of genome editing in different countries appear to be a reflection of a social transformation around food which values natural products. Some have argued that genome editing should be distinguished from other new agricultural technologies such as the generation of transgenic plants by recombinant DNA (gene-splicing) techniques. This argument may be based on the fact, as mentioned above, that with genome editing, only a few nucleotides of a plant genome sequence may be altered (and, therefore is more natural), while transgenesis introduces genes from other species, such as viruses, bacteria, or eukaryotes. As such, the discussions of these issues become almost theological in nature, not unlike debates overhow many angels can dance on the head of a pin, rather than based on science.

Some observers believe that the concept of cisgenesis (as opposed to transgenesis) could be a way to assuage or minimize the concerns that some people have about genetic engineering with the newer, more precise techniques. It refers to the genetic engineering of a recipient plant with genes from a crossablesexually compatibleplant. The process adds no new genes or sequences not found in a compatible plant, and also absent are all selectable marker sequences such as antibiotic resistance or luciferase (which makes a recipient light up), whose presence in transgenic plants is often problematic for anti-GMO activists. Cisgenesis is sometimes proposed as a way to accomplish rewilding, that is, reintroducing into crop varieties desirable properties such as resistance to pathogens or drought present in wild relatives.

History is instructive. Humans have been selecting and breeding to introduce or enhance desirable traits such as yield or taste for millennia, but because of the imprecision of the techniques, this has often led to various beneficial wild genes eventually, inadvertently being bred out and lost over time. Precision and predictability are important to ensure that the results are safe and achieve their desired ends. There are notable historical examples of the use of older, pre-molecular techniques of genetic modification in agriculture that turned out to be problematic. Examples include theLenape potato, which contained elevated, harmful levels of a plant alkaloid; the creation of hyper-aggressiveAfricanized honeybeesby crossbreeding African and European species in the 1950s; and inadvertently causing some varieties of corn in the United States to becomemore susceptible to the Southern Corn Leaf Blight fungus, which resulted in significant crop losses in 1970.

We emphasize that cisgenesis is aresult, not a technique or technology. Only selected genes are introduced into the cultivar, but not unwanted genes that may be responsible for toxicity or other undesirable traits such as bad taste or lower yield. The easiest way by far to accomplish this is by the use of molecular techniques, such as recombinant DNA technology or gene editing.

If plant breeders try to rewild crops using conventional approaches, they are often faced with linkage drag, in which unwanted, sometimes deleterious genes get passed along with the desired trait. That then requires successive generations of recurrent backcrossing and simultaneous selection to create a cultivar in which the gene of interest is no longer linked to any undesirable genes. This can be a long and slow process, taking many years, depending upon how tightly linked the genes are and the generation time of the plant.

By contrast, cisgenesis isolates only the gene(s) of interest from the donor wild plant, which makes it possible to produce disease-resistant trees, such as apples resistant to fire blight fungus disease, for example, or potatoes resistant to late blight disease. It is particularly appropriate for the lengthy process of tree breeding, as well as for producing vegetative crops such as grape, potato, or banana. Cisgenesis can also facilitate the stacking of resistance genes from several sexually compatible plants, in order to introduce resistance to multiple threats. Also, because the sequences introduced into cisgenic crops are derived from plants that are sexually compatible, the resulting plants are indistinguishable from their traditionally bred counterparts. They contain no foreign sequences, thanks to the use of enzymes that remove selectable marker genes.

Research studies of European consumers acceptance of cisgenic crops have been encouraging, and some have argued that if the category of cisgenic crops, whatever the techniques used to craft them, were deemed acceptable for cultivation by European regulators, that could cause a paradigm shift in regulation. In other words, cisgenics would be a kind of Trojan Horse, opening the way to future deregulation. Thus, there is amovement in some quartersto regulate cisgenic plants crafted with molecular techniques no differently than similar plants made with conventional techniques. Health Canada is expected to announce that policy any day. But even with our current state of knowledge about the seamless continuum of techniques of genetic modification with respect to risk, by regulating gene-edited crops like conventionally modified ones, their food regulators create a meaningless distinction between cisgenic gene editing and transgenic, recombinant DNA modifications.

We find that approach flawed and unpersuasive, because it is unscientific. The mere fact of transgenesis the process of introducing atransgene(i.e. an exogenous gene) from one organism into another so that it exhibits a new, heritable property isunrelated to risk. Moreover, genomic sequencing has revealed that extensive DNA changes occur during conventional breeding, and that some plants such as sweet potato have, over time, incorporated into their own genome fragments of DNA from the pathogenic microorganism Agrobacterium and are thus, in effect, natural GMOs.

Simply stated, whether it encompasses gene editing or not, GMO is an arbitrary and meaningless pseudo category, and regulating it more stringently than conventional breeding makes no sense. Once we spurn science as the basis for regulatory policy, weve relegated ourselves to a game of, How stupid and irrational do we intend to be? In other words, where is the limit on accepting false assumptions?

The science tells us that there is a seamless continuum of genetic modification processes from the natural selection that occurs as the result of Darwinian evolution, including the natural GMOs alluded to above; selection and breeding; mutagenesis; somaclonal variation; wide crosses; recombinant DNA; and gene editing. But many national regulatory agencies continue to ignore this and impose oversight regimes that discriminate against the use of the most precise and predictable techniques, exactly the opposite of what risk analysis dictates. Although a strategy of favoring cisgenics might result in some genetically engineered plants being cultivated in Europe and other countries with stringent regulation of those superior techniques, it would further stigmatize transgenic crops. It would represent expediency over principle.

How do we resolve this regulatory conundrum? There have, in fact, been workable models of scientifically defensible, risk-based approaches to regulation (such as hereandhere), but they have not been widely or comprehensively adopted. Even so, we take the long view that sound science must prevail in crafting regulatory policy. Many of us in the scientific community will settle for nothing less. Forsaking science does not end well.

Henry Miller, a physician and molecular biologist, is a senior fellow at the Pacific Research Institute. He was a Research Associate at the NIH and the founding director of the FDAs Office of Biotechnology. Find Henry on Twitter @henryimiller

Kathleen Hefferon, Ph.D., teaches microbiology at Cornell University. Find Kathleen on Twitter @KHefferon

A version of this article was originally posted at Human Events and has been reposted here with permission. Human Events can be found on Twitter @HumanEvents

Excerpt from:
Viewpoint: Canada poised to join expanding number of countries endorsing crop gene editing. That's encouraging but global reform remains elusive -...

As part of the European Union, the UK was a stronghold of anti-GMO opposition. Post-Brexit, however, Britain is changing its outlook for the better. After more than a year of intense public debate, the country'sDepartment for Environment, Food & Rural Affairs (DEFRA) recently announced less restrictive rules that will help the UK's biotech sector develop gene-edited crops designed to boost sustainable farming:

The rule changes, made possible by the UKs departure from the EU, will mean that scientists across England will be able to undertake plant-based research and development, using genetic technologies such as gene editing, more easily.

The rules will apply to plants where gene editing is used to create new varieties similar to those which could have been produced more slowly through traditional breeding processes and will unlock research opportunities to grow crops which are more nutritious, and which require less pesticide use.

This is a tremendous step in the right direction for a country that has denied farmers the benefits of crop biotechnology for decades. Conspicuously missing from DEFRA's announcement, though, was any reference to transgenic crops, the wrongly maligned "GMOs" we're all familiar with. [1] While transgenic technology could benefit UK farmers and consumers, as it has in dozens of other countries, regulators remain unwilling to take on the politically charged fight that would precede the reformation of Britain's GMO regulations, at least for now.

In the spirit of hastening the UK's acceptance of all crop biotechnology, I recently partnered with the London-based Adam Smith Institute to produce a new report titled Splice of Life: The case for GMOs and gene editing. In it, I survey more than two decades' worth of research documenting the benefits of growing and consuming GMOs. The key takeaways are as follows:

How could the UK so liberate its biotech industry? I argue that the ideal regulatory framework is a case-by-case risk assessment that evaluates each novel organism based on the harms it may pose to humans and the environment, regardless of which breeding method produced it. The organisms characteristics and intended use would determine the degree of scrutiny applied by regulators.

Matt Ridley, legendary science writer and member of the UK's House of Lords, had this to say about Splice of Life:

The governments sluggishness in embracing gene engineering is disappointing. This technology, in which Britain could be world-leading, provides immense benefits to farmers, consumers and the environment. Yet, as this important new report from the Adam Smith Institute highlights, gene editing will be severely hampered and GMOs will be left behind. Scientific evidence, not activist superstition, should be at the centre of policy making.

[1] For the record, "GMO" is a nonsense term no scientist uses in a professional context. Nearly all food crops we consume were the products of traditional plant breeding, which "genetically modified" them in all sorts of ways. Transgenic plants are not unique in this respect.

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World Tour: ACSH Makes The Case For GMOs In The UK - American Council on Science and Health