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Posts Tagged ‘CRISPR

Gene editing- Regulatory and ethical challenges

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By: Chringma Sherpa, Ph.D.

Image by Colin Behrens from Pixabay 

When power is discovered, man always turns to it. The science of heredity will soon provide power on a stupendous scale; and in some country, at some point, perhaps, not distant, that power will be applied to control the composition of a nation. Whether the institution of such control will ultimately be good or bad for that nation, or for humanity at large, is a separate question.

William Bateson, English biologist who coined the term “genetics.”

On November 25, 2018, in an allegedly leaked YouTube video, He Jiankui, a scientist at the Southern University of Science and Technology in Shenzhen, China, revealed the birth of the first gene-edited babies using a technology called CRISPR. There has been a general consensus in the scientific community that heritable changes should not be made to prevent the off-target and unwanted genetic changes artificially produced in an individual during gene editing to be passed on to his/her offspring(s). He became the first scientist to publicly violate this consensus resulting in an international scandal and criminal/ethics investigations into both He and his collaborators.

In the wake of He’s CRISPR-babies scandal, scientists worldwide are debating on the ethical and regulatory measures that would discourage another wayward and rogue scientist like He from attempting such an irresponsible feat.  At the 2nd international summit on human gene editing that convened two days after He’s video became public, He presented his work. The summit was well attended by ethicist and journalist besides scientists. At the summit, David Baltimore of the California Institute of Technology, who chaired the organizing committees for both the 1st and 2nd international summits on human gene editing read one of the conclusions from the 1st summit held at Washington DC in 2015 – “It would be irresponsible to proceed with any clinical use of germline editing unless and until (i) the relevant safety and efficacy issues have been resolved, based on appropriate understanding and balancing of risks, potential benefits, and alternatives, and (ii) there is broad societal consensus about the appropriateness of the proposed application”. Baltimore called He’s work outright irresponsible on the basis of the statement from the 1st summit. At the summit, many other ethical and safety-related questions were raised which He failed to answer or did not answer convincingly. 

He’s scandal has driven various organizations to draft new guidelines and sanctions aimed at preventing unethical and unapproved use of genome editing.  China has imposed new laws requiring human gene editing projects to be approved by China’s health ministry first to avoid fines and blacklists. Both the 2nd human gene editing summit and the WHO panel that convened in March 2019, have proposed a central registry of human gene-editing research and called for an international forum/ committee to devise guidelines for human gene editing based on common norms and differences of opinions between countries.  To allow time for the creation and effective implementation of new regulations, the WHO also called for a global moratorium on heritable editing of human eggs, sperm, or embryos for the next five years. Supporting the WHO panel’s recommendations, Francis Collins, director of the National Institute of Health, said that “NIH strongly agrees that an international moratorium should be put into effect immediately”. However, not all scientists are in favor of a moratorium, as they believe it might stifle the growth of a technology that might be safe and beneficial in the near future. Jennifer Doudna of the University of California, Berkley, one of the co-inventors of CRISPR gene editing, says that she prefers strict regulation that precludes the use of germline editing until scientific, ethical, and societal issues are resolved over a moratorium. David Baltimore agrees with Doudna stating that the word moratorium was intentionally not used in both the human gene editing summits as a moratorium would be hard to reverse.  Science historian Ben Hurlbut of Arizona State University, who had numerous discussions with He before Lulu and Nana were created, thinks a blanket moratorium on clinical germline editing would have prevented He from proceeding. Both the two human gene editing summits and a 2015 essay by Baltimore, Doudna, and 16 co-authors had already outlined numerous guidelines for clinical germline editing. According to Hurlbut, He weighed these criteria and believing that his procedure met all the guidelines proceeded. A categorical prohibition of germline editing would not have allowed him to use his subjective judgment and act out of self-interest. 

The modern debate over CRISPR editing is not the first time the scientific community has come together to discuss game-changing biological technologies, and it is heavily informed by two prior events. In 1970, Paul Berg and his postdoctoral researcher David Jackson used the recombinant DNA technology to create the first chimeric DNA. This invention created an uproar among the scientists and the general public who feared that this technology would lead to the creation of uncontrollable and destructive superbugs, the exaggerated versions of which can be seen in some science fiction movies. Yielding to the opinions and sentiments of the fellow scientists, Berg held himself from cloning such recombinant DNAs and in 1974, he pleaded for a voluntary moratorium on certain kinds of recombinant DNA research until their safety issues have been resolved.  He also moved quickly to organize the Asilomar conference (Asilomar II) in 1975 that bore semblance to the 2nd human gene editing conference in that it invited not only the scientists but lawyers, ethicists, writers, and journalists to weigh in on the risk-benefit analysis of the Recombinant DNA technology. On the recommendation of Asilomar conference, Donald Fredrickson, then director of the National Institutes of Health (NIH), initiated the formation Recombinant DNA Advisory Committee (RAC) to act as a gatekeeper of all research that involved recombinant DNA technology. The scope of the committee, which was composed of stakeholders, including basic scientists, physicians, ethicists, theologians, and patients’ advocates was later expanded to encompass the review and approval of human gene therapy research. Due to the redundancies of regulatory oversights between the US Food and Drug Administration (FDA) and RAC, RAC was reinstated as only an advisory body providing advice on the safety and ethical issues associated with emerging biotechnologies in 2019.

While this is a successful example of scientific self-regulation, the second event resulted in a major setback in the field of gene therapy. On September 13, 1999, Mark Batshaw and James Wilson of University of Pennsylvania supervised the administration of adenovirus to an 18-year-old Jesse Gelsinger in a gene therapy clinical trial. Gelsinger died of liver and kidney failure and brain damage three days later. Like the birth of CRISPR babies, Gelsinger’s death was an instance where new technology was used prematurely without a thorough assessment of its safety profile. It is suspected that both the clinical applications headed by He and Wilson might also have been motivated by fame and financial gain; He and Wilson both had financial stakes in private biotechnology companies that would benefit from these human trials. In the aftermath of Gelsinger’s death, Wilson was banned from carrying out FDA regulated clinical trials for the next five year, nearly all gene therapy trials were frozen, and many biotechnology companies carrying out these trails went bankrupt. This was a dark period in the history of gene therapy, and it would take almost another decade of introspection, reconsideration, and more basic experimentation for gene-therapy to re-emerge as a viable therapeutic strategy.

Figure 1: The regulatory status of human germline gene modification in various countries. Thirty-nine countries were surveyed and categorized as “Ban based on legislation” (25, pink), “Ban based on guidelines” (4, faint pink), “Ambiguous” (9, gray), and “Restrictive” (1, light gray). Non-colored countries were excluded in this survey. Adapted from Araki, M. and Ishii, T (2014): “International regulatory landscape and integration of corrective genome editing into in vitro fertilization” Reproductive Biology and Endocrinology, 2014 12:108

Scientists at both the Asilomar and human gene editing conferences passionately debated the safety of the relevant technologies but deliberated on the discussion of the big ethical issue associated with these technologies – the ultimate creation of designer babies. That gene editing sits on the slippery slope to eugenics was recognized since the days of Charles Darwin and Gregor Mendel when the study of genes and heredity was still in its infancy and the discovery of DNA as the genetic material was half a century away. One of the earliest proponents of genetic manipulation for human benefits was Francis Galton, Charles Darwin’s cousin. Galton proposed an unnatural and accelerated selection of beneficial traits by marriage between people of desirable traits. The danger that someday some rogue scientists might use germline gene editing technology in favor of eugenics lurks in the mind of those who understand the potential of the currently available gene editing technologies. However, more fearful is the idea that the wave of positive eugenics would soon give way to negative eugenics – elimination of undesirable traits as it did around World War II as exemplified by the famous case of Carrie Buck, a woman who was designated “mentally incompetent” and involuntarily sterilized. 

Various countries have their own regulation and legislation on germline editing to prevent any backlash from this powerful technology. Figure 1 presents a summary of the regulatory landscape of germline gene modification surveyed in thirty-nine countries by Araki Motoko and Tetsuya Ishii.  In the US, Congress has shown strong support against germline gene editing. In 1996, it passed a rider as part of the annual appropriations bill that prohibits the use of federal funds for any research involving human embryo. In another appropriations bill passed in 2015, Congress banned the FDA from considering applications involving the therapeutic modification of the human germline. 

Human gene editing holds great promises in treating many life-threatening and previously intractable diseases. Only when this discipline of science is held to high ethical standards and regulated sensibly at international, national, and a personal level, shall we reap the benefits of this powerful technology.

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May 29, 2019 at 9:25 am

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Science Policy Around the Web – May 3rd, 2019

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By: Allison Dennis B.S.

Image by Ashley Smith from Pixabay

A More Humane Livestock Industry, Brought to you by CRISPR

Innovations sparked by germline editing in livestock are poised to help farmers make rapid improvements in the care and treatment of their animals by helping overcome inefficiencies genetically. Livestock industries use animals specifically bred to maximize their use and often require or prefer animals of a particular gender. Male cattle produce more meat. Female cows produce milk. Female chickens lay eggs. Known as “the dirty secret” of the dairy industry, farmers under financial pressure may kill male calves soon after birth to avoid the costs of raising them further. Every year, 6 billion male chicks, which hold no economic value for egg production, are killed within a day of hatching. The egg industry is seeking to prevent this, and in June of 2016, the United Egg Producers, a group that represents more than 95% of egg production in the United States, stated their goal of “eliminating the culling of day old male chicks by 2020 or as soon as it is commercially available and economically feasible.”

Scientists are racing to offer these industries solutions by harnessing the power of the gene editing technology CRISPR to genetically engineer animals to break the 50-50 gender rule. Mark Tisard, a geneticist at the The Commonwealth Scientific and Industrial Research Organization, Australia’s national science agency, is using CRISPR to engineer male eggs to glow red under fluorescent light, allowing farmers to repurpose male eggs for vaccines or feed production before the chick has developed. Across the world, the startup eggXYt in Israel is taking a similar approach, making male eggs glow yellow. Alison Van Eenennaam, an animal geneticist at UC Davis, is exploring the possibility of using CRISPR to produce all male herds of beef cattle from the point of conception.

While pre-determining gender remains a major goal, gene-editing has already been used to introduce a gene for slick hair that helps cattle handle heat better in warm climates. Scientists have successfully used CRISPR to produce pigs that are resistant to the PRRS virus, which causes respiratory disease and reproductive failure, costing farmers in the United States half a billion dollars a year. 


The reception of these technologies in the United States remains unclear. Most recently, in January 2017, the FDA drafted rules that would put genetically altered animal products through the same safety and efficacy testing undertaken for new drugs. However, since then, the USDA has clarified that plants produced through genome editing will not be subject to additional regulation if they could conceivably been developed through traditional breeding methods. Those looking to harness the power of these types of genetic modifications for livestock are hoping to see this attitude expanded to animal products.

(Gregory Barber, Wired Magazine)

House Democrats move to resurrect Congress’s science advisory office

At the helm of American society is a group of appointed representatives racing to keep abreast of a deluge issues stemming from technological innovation. A draft of the 2020 Legislative Branch funding bill that was approved by its subcommittee on April 30th offers potential relief in the form of re-establishing the Office of Technology Assessment (OTA). The OTA was originally established in 1972 to provide congressional staff with deep and comprehensive analysis of technical issues to aid in the crafting of public policy, independent from lobbyists and activists. At that time, Anti-Ballistic Missiles and Super Sonic Travel were the technologies stirring up media frenzies. However, the OTA was eliminated in 1995 to reclaim $21 million for the congressional budget. The $3.9 billion dollar proposed budget for 2020 included only $6 million for its revival.

Currently, the Congressional Research Service (CRS) is tasked with keeping Congress informed on technical issues ranging from gene editing to artificial intelligence. In January 2019, recognizing the need for greater technical assistance, the US Government Accountability Office (GAO) established the Science, Technology Assessment, and Analytics team to conduct technology assessments and provide technical assistance directly to Congress. The proposed budget would support these efforts by providing CRS and the GAO an additional $24 million and $26 million respectively. However, some believe that there is still a need for the OTA in spite of these new teams. Representative Sean Casten testified to fund the OTA as he believes there is still a need to “fulfill the forward looking, deep-dive, technical assessments of new scientific and technological developments once provided by OTA.” Representative Mark Takano also expressed support, stating “In the ecosystem of legislative support organizations, OTA plays a unique role. No other entity has the capacity and expertise to provide in-depth and forward-looking analysis of complex, technical issues informed by an understanding of how Congress works.” 

Former Congressman Rush Holt similarly tried to revive the OTA in 2015, only asking for 2.5 million dollars, but his amendment was defeated 248 to164. The 2020 funding bill next heads to the full House Appropriations Committee for markup before representatives get a chance to vote.

(David Malakoff, Science)



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May 3, 2019 at 11:20 am

Science Policy Around the Web – March 15, 2019

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By: Allison Dennis. B.S.

Image by Gabriela Sanda from Pixabay 

A Genetic Mutation Might Explain Why Birth Control Can Fail

Hormonal contraceptives have been increasingly embraced as an effective tool for family planning since their approval for contraceptive use by the FDA in 1960Fewer than 1 in 100women will get pregnant in a year when using the contraceptive pill correctly, but the cause of the few unintended pregnancies remaines a mystery. 

To ask if there was a genetic reason why hormonal contraceptives might be ineffective at preventing pregnancy for some women, researchers measured the amount of etonogestrel in the blood of women using the etonogestrel contraceptive implant (Nexplanon). While the findings of this small study were not significant, the researchers did observe that women carrying a mutation in their CYP3A7gene had lower concentrations of etonogestrel in their blood. Of the women with mutation, 28% had etonogestrel levels lower than what is needed to suppress ovulation. Only 9% of women without the mutation had critically low levels. The CYP3A7 gene produces a fetal enzyme, responsible for breaking down excessive steroid hormones in the developing fetus. Usually the gene is turned off in adulthood, but genetic mutations can result in it staying active throughout one’s life, possibly leading women with these mutations to break down the etonogestrel being supplied by the Nexplanon implant. 

To begin capturing the ability of particular genetic variants to predict which patients will or will not respond to particular medication, experience adverse events, or need a particular dose, the FDA maintains a list of drugs whose use may be complicated by particular Pharmacogenomic Biomarkers. Only one of the 232 on the list is a hormonal contraceptive, listed because women carrying a particular mutation may respond negatively to one of its inactive ingredients, not because it is less effective for these women. The etonogestrel study is the first to associate particular genetic variants with birth control performance. As researchers delve into the complicated intersection between genetic differences and drug responses their discoveries promise to lead to better outcomes for all those seeking effective family planning options. 

(Megan Molteni, Wired Magazine)

New call to ban gene-edited babies divides biologists

The birth of genetically modified children is now possible thanks to technologies like the genome-editing CRISPR-Cas9which can introduce heritable changes in germline cells, including human sperm, eggs, and embryos. In November 2018, He Jiankui’s demonstrated the technique by orchestrating the birth of two babies whose genomes were altered between in vitro fertilization and implantation. He Jiankui’s experiment may have shown editing the human germline was possible, but it did not address the safety or efficacy of the genetic changes for his two inaugural patients. While many view He Jiankui’s experiment as prematureand possibly illegal, it has undeniably brought discussions of the appropriate use of the technology to a forefront. 

On March 13th, a group of prominent scientists and international specialists published a call for a global moratorium on clinically making genetically modified children. The first step, as they see it should be setting a deadline before which all clinical use of germline editing will be put on hold. They suggest five years from now. During this time, they propose that nations engage in “discussions about the technical, scientific, medical, societal, ethical, and moral issues that must be considered.” Technical issues arise from the need to demonstrate germline editing as safe and efficacious. Scientifically, the long-term effects of specific genetic enhancements or corrections must be understood. Many potential changes imagined to be possible may not be medically necessary. As for societal, ethical and moral considerations, the authors stress the importance of carefully weighing “the appropriateness of altering a fundamental aspect of humanity.” The commentary acknowledges that not all nations will be ready to proceed at the same rate but suggests that any nation that does decide to proceed only do so after consulting broadly with other countries and allowing their public to reach societal consensus that the next step is appropriate. 

The motivation of the authors’ is clear. “We’re trying to force the spotlight on what comes next,” said co-author Eric Lander, President of the Broad Institute. To what extent international consensus can be built surrounding what the National Institutes of Health calls“a crucial moment in the history of science: a new technology offers the potential to rewrite the script of human life”  remains to be seen.

(Jon Cohen, Science Magazine)

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March 15, 2019 at 4:00 pm

Science Policy Around the Web – December 18, 2018

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By: Janani Prabhakar, Ph.D.

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Source: Pixabay

How will we outsmart A.I. Liars?

Click bait, such as the new SNAP_R bot, is becoming increasingly sophisticated. Researchers at ZeroFOX, a security company in Baltimore, designed SNAP_R to fool users to click on links in tweets that could lead to hazardous sites. While this was just a proof of concept and did not actually lead users to such sites, the findings are striking about human click behavior: SNAP_R elicited a click 66% of the time, on par with hand-crafted phishing attempts. The bot was designed using neural networks that learned to write phishing messages through tweets, Reddit posts, and other online hacks. Improvements in machine learning methods will allow these types of networks to become smarter and smarter and learn from larger amounts of data that include human specific behaviors through text analyses. Thus, a more powerful network can take advantage of the nuances of human behavior to manipulate innocent users much easier. There are examples of this already in place that are not nefarious, such as Apple’s Siri or Amazon’s Alexa. These platforms use neural networks to learn and decode voice data and generate sounds as well as images in response. Another example is Google’s Duplex system,  an A.I. that can call restaurants and make reservations for you. With advances in language systems, future bots can respond in a more nuanced, almost unrecognizable (as A.I.) manner. Experts believe that with the many global advances in A.I., “we face a machine-learning arms race.” While there are many advances in these types of networks, we are still figuring out methods to detect those that are malicious. The current standard is that of individual responsibility, or the “onus is on the individual” approach. In essence, each person should view every bit of news from an online source with distrust. While at present this is the best standard, it is less easy to control and measure than algorithms that could detect pernicious machine learning algorithms. While these are currently being developed, It will be some time before they can be implemented. The problem is, undoubtedly, complex.

(Cade Metz, New York Times)

 

China orders medical team to stop gene-editing project

Late in November, Dr. He Jiankui announced that he had used CRISPR to change the genomes of twin girls born through IVF. This news made headway and received significant criticisms from universities and government organizations across the world. On November 29th, China’s government ordered a halt to this approach by the medical team, stating with a large global consensus that it was too early to make permanent changes to DNA. Gene editing holds promise for helping individuals with genetic illnesses and studies are currently underway to modify the genome using CRISPR in these individuals. However, little is known about the risks of altering DNA in eggs, sperm, or embryos. To do so outside of laboratory research has been described as a huge violation of research ethics. Currently, Dr. Jiankui plans to release all his raw data for independent verification. This research has brought to light questions about the acceptable risks in conducting research with high impact. There are standard, clear ethical guidelines that many argue Dr. Jiankui should have known he was in violation of. Recently, the journal Nature named Dr. Jiankui in its top 10 people who mattered in science in 2018 list because of the important questions about morality and ethics his work brings to the scientific arena. The topics under discussion include how researchers evaluate the ethical considerations and the policies in place that regulate those considerations – should these be re-evaluated?

 

(Marilynn Marchione, The Christian Science Monitor)

 

 

 

 

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December 18, 2018 at 4:52 pm

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Science Policy Around the Web – December 6, 2018

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By: Neetu M. Gulati, Ph.D.

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The CRISPR Baby Scandal Gets Worse by the Day

Ethical concerns and controversy came to the forefront last week when news broke that Chinese scientist He Jiankui had supposedly created genetically edited babies using CRISPR technology: a first in the world. CRISPR/Cas9 technology, or CRISPR as it is more commonly known, is a scientific tool that allows researchers to edit (add, subtract, or change) the expression of genes quickly and precisely. This technology could be used to fix mutations that cause human disease. However, there are also risks, using CRISPR for gene editing may have disastrous side effects such as potentially leading to cancer.

He Jiankui claimed that he used the technology to alter a gene called CCR5 to reduce the risk of of HIV infection in embryos before implanting them in a woman, who then gave birth to twin girls. He claimed another CRISPR baby may be on the way from another pregnant woman. It is unclear if He has actually done what he claimed, and he has not yet published his results in a peer-reviewed journal. Nevertheless, the response to He’s claims have been strongly negative. Many people are concerned that He violated ethical norms by editing human embryos, especially because the overall consensus among scientistsin the field of gene-editing was that “there is a need for caution” and to only use the technology after “much more research to meet appropriate risk/benefit standards.”

Since the public has learned about He’s experiments, numerous scientists, including pioneers in the CRISPR field, have spoken out against He’s actions and have called for a temporary moratorium on similar experiments. Southern University of Science and Technology in China, where He has been on unpaid leave since February, has opened an investigation into He after finding out about his research. China’s National Health Commission is also investigating He.

Amid the backlash, He defended himself and his actions at the Human Genome Editing Summit in Hong Kong, claiming to be “proud” of his work. Dr. He has not been seen since the summit, however, and there are now concerns that he may be missing.

(Ed Yong, the Atlantic)

 

Trump emphasizes workforce training in new vision for STEM education

The White House released a new five-year strategic plan for science, technology, engineering, and mathematics (STEM) education this week, with a vision that “all Americans will have lifelong access to high-quality STEM education and the United States will be the global leader in STEM literacy, innovation and employment.” The report emphasizes workforce training in STEM, focusing primarily on opportunities outside of traditional classroom settings, such as apprenticeships. The plan also highlights the need for more diversity in STEM, such as minorities and women.

Overall the strategic plan focuses on four pathways to success: developing and enriching partnerships between educators, employers, and the community; engaging students in trans-disciplinary learning, including advancing innovation and entrepreneurship education; building computational literacy; and operating with transparency and accountability. The plan put forth by the Trump administration diverges from some of the key priorities of the former administration, including efforts focused on traditional academic environments such as training more teachers, and improving STEM instruction in colleges and universities. Instead, this plan appears more focused on how STEM education prepares students for the years after schooling is completed. “STEM education is absolutely critical to supporting the American worker, and this plan brings together a number of programs that are part of our emphasis on the American worker,” said Michael Kratsios, deputy assistant to the president at OSTP.

(Jeffrey Mervis, Science)

 

NASA’s InSight Mars explorer lands safely on the Red Planet

For only the eighth time in human history, a spacecraft has been landed on Mars. The InSight lander touched down on Martian soil on November 26, 2018 after over six months of space travel.

NASA’s InSight mission aims to gather information about Mars, and is part of the NASA Discovery program for focused solar science missions. InSight will study the crust, mantle, and core of Mars, to allow scientists to learn more about the formation of rocky planets in the solar system.

InSight has already begun taking photos of the surface of Mars, which have been posted on social media accounts such as Twitter. The lander has also set up solar panels, which allows InSight to power its cutting edge instruments. In doing so, the lander set an ‘off-world record,’ generating more electrical power than any previous vehicle on the planet’s surface. InSight project manager Tom Hoffman spoke on the importance of this achievement, “The 4,588 watt-hours we produced during sol 1 means we currently have more than enough juice to perform these tasks and move forward with our science mission.” This almost doubles the energy produced in a Martian day produced by NASA’s Curiosity rover, which previously held the record.

InSight will continue taking pictures of the surface of Mars to study its new surroundings and use its robotic arm to set up instruments to place them on the surface of Mars for the next few weeks. It will take two to three months before the lander begins conducting science on the Red Planet.

 

(Sarah Kaplan, Washington Post)

 

 

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December 6, 2018 at 5:23 pm

Science Policy Around the Web – November 27, 2018

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By: Allison Dennis, B.S.

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Source: Pixabay

 

California’s Wildfires Could Mean A Generation Of Lung Problems

The acute dangers of uncontrolled wildfires are undeniable, yet chronic dangers remain poorly understood. Changes in air quality due to wildfire smoke may have long-term and widespread health effects that researchers are only beginning to decipher. In 2017, nature provided the near perfect conditions for a much needed experiment. As the Mendocino Complex Fire raged, its smoke drifted over 200 miles to blanket the living space of an outdoor colony of primates bred for research for 10 days. 500 infant rhesus macaques, a commonly used model of human disease, were exposed, allowing respiratory immunologist Lisa Miller to begin an experiment looking for long-term respiratory damage in a pediatric population. Her previous studies of a smaller group of monkeys exposed in 2008 revealed monkeys born in wildfire conditions grew up to have a reduced lung capacity and compromised immune system . Ten years after the fire, monkeys who were infants at the time of the fire have a high incidence of idiopathic pulmonary fibrosis, a fatal human disease associated with environmental pollutants and cigarette smoking. By carefully recreating her impromptu 2008 experiment, Miller is hoping to gain deeper insights into what damage can occur in the developing lung tissue that will lead to possible interventions.

Miller’s research already suggests that a brief exposure to smoke early in life can have a lifetime of consequences. Smoke inhalation is much more widespread than the immediate dangers of fire, and will need to be incorporated into disaster preparedness plans. Financial assistance may be needed to help families temporarily relocate following fires not only due to burned homes, but also to homes blanketed in smoke. Currently, websites like airnow.gov provide up to date measures of air quality and can be used to decide when to limit children’s time outdoors. Parents with other options may need to weigh the potential risks of raising a family in places where wildfires are an annual occurrence.

(Maggie Koerth-Baker, FiveThirtyEight)

 

Genome-edited baby claim provokes international outcry

 

The announcement of the first genome-edited babies is shocking ethicists, scientists, and spectators around the world. He Jiankui of the Southern University of Science and Technology of China in Shenzhen claims to have altered the embryonic DNA of two twin girls born in November 2018 by using CRISPR-cas9 to disable the protein CCR5, known to provide HIV access to human cells. The study has not yet been submitted for publication and will likely undergo extensive peer review for verification. He recruited couples looking to conceive, where the male partner had HIV. The risk of transmission of HIV between father and offspring is very low, and removing semen from sperm before fertilization is commonly used to further mitigate the small risk. However, He thought these would-be parents would especially value the benefits of conferring their child with a lifetime protection against HIV through altered genetics.

Since its first demonstration as a gene-editing technique nearly ten years ago, ethicists have debated the potential application of CRISPR-cas9 to alter human DNA. Because reproductive tissues develop from the edited zygotic cells, it is likely that the twins will pass these changes on to their offspring along with any other possible off-target changes to the genome. While lacking the CCR5 gene has been shown to confer protection against HIV infection, it may subsequently increase the risk of other viral infections. Many still feel that not enough is known about the long-term and generational effects of altering a person’s germ-line using these proteins to justify the risks they could pose over an unborn person’s lifespan. To what extent the institutions that facilitated the experiment were consulted or informed of the true nature of the search remains unclear. The trial was only registered on November 8th, well after the work had begun. However in the race to be first, He appears to understand that any harm to the children arising from the experiment stating these risks are “going to be my own responsibility.”

 

(David Cyranoski, Nature News)

 

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November 27, 2018 at 10:24 am

Science Policy Around the Web – September 11, 2018

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By: Liu-Ya Tang, Ph.D

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New Therapies

Here’s what we know about CRISPR safety – and reports of ‘genome vandalism’

Since its discovery in 2012, CRISPR-Cas9 has become a popular gene-editing tool forr removing, adding or altering sections of the DNA sequence in the genome. It has been widely used in research settings because it is efficient, precise and cheap. Beyond that, there is a growing interest in applying CRISPR techniques in treating human diseases which are caused by genetic mutations. Firms such as CRISPR Therapeutics, Intellia Therapeutics and Editas Medicine are conducting clinical trials of therapies using CRISPR. For example, one of the pipelines from CRISPR Therapeutics is a gene-editing Phase I/II trial for beta-thalassemia and sickle cell disease, which are caused by mutations in β-globin gene. Hematopoietic blood cells, which are the stem cells that generate red blood cells, are taken from the patient. After genetic correction, the cells are then reintroduced to the same patient. The hope is that the “revised” stem cells will produce normal red blood cells, which will cure the disease, relieving the patients from a lifetime of blood transfusions. Furthermore, there are other clinical trials of treatments for Hemophilia, Duchenne muscular dystrophy and cystic fibrosis by CRISPR. In China the technology has been used to treat patients with oesophagus cancer.

Though there is great potential for applying CRISPR to cure human diseases caused by various genetic mutations, cautionary measures need to be implemented while the safety of this technology is being debated. Several studies have raised concerns that CRISPR may not be as effective as previously thought, and in some cases it may produce unwanted side effects. The data have shown that applying CRISPR-Cas9 system on human body may cause stress to cells and some people may not be responsive to CRISPR-based therapy if their immune system has developed an immune defense against the CRISPR protein. Additionally, studies show that CRISPR-Cas9 may cause off-target DNA damage during genome editing, and the rate is about 1 to 5 percent. However, the clinical application of CRISPR editing is a work in progress. Hopefully new versions of the technique with improved accuracy and efficiency will be used to treat genetic diseases in the near future.

(Jianhua Luo, The Conversation)

Public health

Hospitals are fed up with drug companies, so they’re starting their own

Shortages of critical generic drugs and price spikes on old drugs have plagued hospitals in recent years. To resolve this, a group of major American hospitals are launching a nonprofit and independent generic drug company, which is called Civica Rx.

The company’s mission is to “make sure essential generic medicines are affordable and available to everyone”, so it will focus initially on establishing price transparency and stable supplies for 14 generic drugs used in hospitals. Civica is backed by seven large health systems and three philanthropic groups including Mayo Clinic and HCA Healthcare, collectively representing about 500 hospitals. A commitment of $100 million from governing members have been contributed to the effort, and member health care organizations have agreed to buy drugs from Civica based on the long-term contracts.

The list of 14 drugs haven’t been disclosed because of competitive reasons, but they include generic drugs that underwent price increases of 50 percent or more between 2014 and 2016 and essential medicines that were on national shortage lists. It is estimated that the first drug from Civica could hit the market next year.

The establishment of Civica would present a “threat” to other pharmaceutical companies that make the same generic drugs. There is a possibility that those companies will temporarily cut their prices to maintain the market share. But Civica leaders say the model of guaranteeing a steady supply at a fair, transparent price will be attractive to hospitals. Martin VanTrieste, chief executive of Civica, hopes that the entrance of 14 generic drugs to the market is a good correction for the over-priced drug market and the company would expand if the market place cannot be fixed by adding these 14 drugs.

(Carolyn Y. Johnson, The Washington Post)

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Written by sciencepolicyforall

September 12, 2018 at 8:56 am