Scientists, ethicists slam decisions behind gene-edited twins

Chinese geneticist He Jiankui speaks during the Second International Summit on Human Genome Editing at the University of Hong Kong days after the Chinese geneticist claimed to have altered the genes of the embryo of a pair of twin girls before birth, prompting outcry from scientists of the field.

As more details regarding the first gene-edited humans are released, things continue to look worse. The researcher who claimed the advance, He Jiankui, has now given a public talk that includes many details on the changes made at the DNA level. The details make a couple of things clear: we don’t know whether the editing will protect the two children from HIV infections, and we can’t tell whether any areas of the genome have been damaged by the procedure.

All of that raises even further questions as to whether He followed ethical guidelines when performing the work and getting consent from the parents. And, more generally, nobody is sure why He chose to ignore a strong consensus that the procedure wasn’t yet ready for use in humans. In response to the outcry, the Chinese government has shut down all further research by He, even as it was revealed that a third gene-edited baby may be on the way.

While the US already has rules in place that are intended to keep research like He’s from happening, a legal scholar Ars spoke with suggested there may be a loophole that could allow something similar here. In light of that, it’s important to understand the big picture He has potentially altered. What exactly happened in China and why does it concern so many in the scientific community?

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Reports out of China suggest first human gene-edited babies have been born

He Jiankui, the scientist who has claimed to have led an effort to gene-edit humans.

On Sunday, news reports indicated that the first gene-edited human babies had been born in China. As of right now, the information on what, exactly, has been accomplished is confusing. The scientist behind the announcement has made a variety of claims but has not submitted his data to the community in order for his claims to be verified. But even in its current state, the announcement has set off a firestorm of criticism within the scientific and ethics communities. Most scientists feel that the technology isn’t ready for use in humans and that there are better ways to deal with the problem the work was addressing: HIV infection.

Editing genes

The most complete report we currently have comes from the Associated Press. Its reporters talked to the researcher behind the announcement, He Jiankui of Shenzhen, China, in advance of his public announcement. That public announcement came at the start of the Second International Summit on Human Genome Editing, taking place this week in Hong Kong. The summit is intended to help work out the “science, application, ethics, and governance of human genome editing,” but He apparently chose to go ahead in advance of those being settled.

He is expected to present more details of his work on Wednesday, but it’s clear that he used biotechnology called CRISPR to perform the gene editing. CRISPR is a system that evolved in bacteria to protect them from viruses by allowing them to recognize and cut viral DNA. By changing part of the CRISPR system, it’s possible to direct it to cut an arbitrary DNA sequence. That can include sequences within the human genome.

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CRISPR scientist in China claims his team’s research has resulted in the world’s first gene-edited babies

In a dramatic development for CRISPR research, a Chinese scientist from a university in Shenzhen claims he has succeeded in helping create the world’s first genetically-edited babies. Dr. Jiankui He told the Associated Press that twin girls were born earlier this month after he edited their embryos using CRISPR technology to remove the CCR5 gene, which plays a critical role in enabling many forms of the HIV virus to infect cells.

The AP’s interview comes after the MIT Technology Review reported earlier today that He’s team at the Southern University of Science and Technology wants to use CRISPR technology to eliminate the CCR5 gene and create children with resistance to HIV. The news also comes the day before the Second International Summit on Human Genome Editing is set to begin in Hong Kong.

According to the Technology Review, the summit’s organizers were apparently not notified of He’s plans for the study, though the AP reports that He informed them today. (It is important to note that there is still no independent confirmation of He’s claim and that it has not been published in a peer-reviewed journal.)

During his interview with the AP, He, who studied at Rice and Stanford before returning to China, said he felt “a strong responsibility that it’s not just to make a first, but also make it an example” and that “society will decide what to do next.”

According to documents linked by the Technology Review, the study was approved by the Medical Ethics Committee of Shenzhen HOME Women’s and Children’s Hospital. The summary on the Chinese Clinical Trial Registry also says the study’s execution time is between March 7, 2017 to March 7, 2019, and that was seeking married couples living in China who meet its health and age requirements and are willing to undergo IVF therapy. The research team wrote that their goal is to “obtain healthy children to avoid HIV providing new insights for the future elimination of major genetic diseases in early human embryos.”

A table attached to the trial’s listing on the Chinese Clinical Trial Registry said genetic tests have already been carried out on fetuses of 12, 19, and 24 weeks of gestational age, though it is unclear if those pregnancies included the one that resulted in the birth of the twin girls, whose parents wish to remain anonymous.

“I believe this is going to help the families and their children,” He told the AP, adding that if the study causes harm, “I would feel the same pain as they do and it’s going to be my own responsibility.”

Chinese scientists at Sun Yat-sen University in Guangzhou first edited the genes of a human embryo using CRISPR technology (the acronym stands for Clustered Regularly Interspaced Short Palindromic Repeats), which enables the removal of specific genes by acting as a very precise pair of “genetic scissors,” in 2015. Though other scientists, including in the United States, have conducted similar research since then, the Southern University of Science and Technology’s study is considered especially radical because many scientists are wary of the ethical implications of CRISPR, which they fear may be used to perpetuate eugenics or create “designer babies” if carried out on embryos meant to be carried to term.

As in the United States and many European countries, using a genetically-engineered embryo in a pregnancy is already prohibited in China, though the Technology Review points out that this guideline, which was issued to IVF clinics in 2003, may not carry the weight of the law.

In 2015, shortly after the Sun Yat-sen University experiment (which was conducted on embryos that were unviable because of chromosomal effects) became known, a meeting called by several groups, including the National Academy of Sciences of the United States, the Institute of Medicine, the Chinese Academy of Sciences and the Royal Society of London, called for a moratorium on making inheritable changes to the human genome.

In addition to ethical concerns, Fyodor Urnov, a gene-editing scientist and associate director of the Altius Institute for Biomedical Sciences, a nonprofit in Seattle, told the technology Review that He’s study is cause for “regret and concern” because it may also overshadow progress in gene-editing research currently being carried out on adults with HIV.

TechCrunch has contacted He for comment at his university email.

Synthego raises $110 million to make gene editing technologies more accessible

Paul Dabrowski, the chief executive officer of Synthego, which provides genetically engineered cells to scientists and researchers, worries about a future where access to the genetic technologies that will reshape the world are only available to the few who can afford them.

To hear him tell it, that’s why Dabrowski began working on Synthego in the first place — to democratize access to the new technologies that will give scientists, researchers, and consumers new ways to rewrite the code that has defined human existence.

“People talk about access to the tools, but the question is access to the therapies,” Dabrowski said. “We’re talking about the basis of what does it mean to be human not right now, but in the next 100 years.”

Now, the company has a fresh $110 million in cash from new investors at Founders Fund and the company’s previous backers — 8VC and Menlo Ventures — to try and drive costs down.

“This new funding allows us to expand our reach and build out of our full stack platform capabilities at a perfect time,” said Dabrowski, co-founder and CEO, Synthego, in a statement. “Biological medicines are on the cusp of a revolution with the coming curative cell and gene therapies, and we are proud to support this industry.”

While Dabrowski said the financing will be used for further research and development — and bringing new services to market — in the near term the funding will be used to expand two main areas of interest for the company. One is the creation of CRISPR kits that can create different genetic lines based on the requests from researchers and scientists, and the other is creating materials that are “clinical-grade”, which means that they can be used in clinical trials on animal (and potentially human) subjects.

“In general the demand for these products is quite high. Building capacity and building out the informatics models for the predictability on the CRISPR research side. 

In all, the Redwood City, Calif.-based company has raised $166 million in funding to develop its technology that makes research and development using the gene editing tool known as CRISPR more economical and faster for researchers. Synthego claims that  by offering researchers one-click access to engineered cells with guaranteed edits in their desired target, the company can slash the time it takes to conduct experiments by months, enabling predictable and rapid outcomes in cell and gene therapy research and development. 

As we’d written previously, Synthego launched its first CRISPR offerings to the market earlier this year.

There are two basic functions that people use CRISPR for, said Dabrowski. The first is to remove a gene or function and the second is adding a function to genetic material.

Both of those processes involve three (very complicated) steps. First scientists have to identify the gene that they want to target and then understand what genetic material within that gene they want to target for removal. Then a research team would need to identify and procure the reagents and components they need to edit a gene. Finally, the team would need to figure out whether the edit was made successfully and watch for results when the edited genetic material is cultivated.

Synthego’s first set of products were designed to simplify the process for identifying and designing genetic material for experimentation. This next set of tools are supposed to help scientists by providing them with the material they want to observe or experiment with.

“Our vision is a future where cell and gene therapies are ultimately as accessible as vaccines, so that everyone can benefit from next-generation cures,” said Dabrowski in a statement. “Synthego will continue to innovate to help researchers redefine the boundaries of transformative medicines.”

HP is ‘printing’ drugs for the CDC to speed up antibiotic testing

At least 2 million people in the U.S. become infected with so-called “super bugs” and at least 23,000 people die as a direct result of these infections each year, according to the Centers for Disease Control (CDC). Now, HP’s Biohacker technology is working with the CDC on a pilot program to “print” and test antibiotics in an effort to catch these antimicrobial resistant strains from spreading faster.

The HP D300e Digital Dispenser BioPrinter technology works by using the same set up as a regular ink printer, but instead dispenses any combination of drugs in volumes from picoliters to microliters to be used for research purposes.

Part of the reason these bugs spread so rapidly often comes down to mis-use of antibiotics, leading the bacteria to develop a resistance to the drugs available. The CDC hopes to give hospital providers access to the technology nationwide to cut down on the problem.

“Once a drug is approved for use, the countdown begins until resistance emerges,” Jean Patel, PhD, D (ABMM), Science Team Lead, Antibiotic Resistance Coordination and Strategy Unit at CDC said in a statement. “To save lives and protect people, it is vital to make technology accessible to hospital labs nationwide. We hope this pilot will help ensure our newest drugs last longer and put gold-standard lab results in healthcare providers’ hands faster.”

The 3D bioprinting sector has been experiencing rapid growth over the last few years and will continue on pace through the next decade, mainly due to R&D, according to market researchers. Innovation in the space includes printing of organs and human tissue and drug research and development.

Further, this potentially valuable antibiotic resistance research could help patient care teams stem a grim future where we experience a regression in health and life spans due to no longer having the ability to treat currently curable diseases.

The HP BioPrinter is currently used by labs and pharmaceutical companies such as Gilead, which tests for drugs used against the Ebola virus. It is also being used in various CRISPR applications. The CDC will use these printers in four regional areas spread throughout the U.S. within the Antibiotic Resistance (AR) Lab Network to develop antimicrobial susceptibility test methods for new drugs, according to HP.

Gene editing crunches an organism’s genome into single, giant DNA molecule

Complex organisms have complex genomes. While bacteria and archaea keep all of their genes on a single loop of DNA, humans scatter them across 23 large DNA molecules called chromosomes; chromosome counts range from a single chromosome in males of an ant species to more than 400 in a butterfly.

There have been indications that chromosomes matter for an organism’s underlying biology. Specialized structures within them influence the activity of nearby genes. And studies show that areas on different chromosomes will consistently be found next to each other in the cell, suggesting their interactions are significant.

So how do we square these two facts? Chromosome counts vary wildly and sometimes differ between closely related species, suggesting the actual number of chromosomes doesn’t matter much. Yet the chromosomes themselves seem to be critical for an organism’s genome to function as expected. To explore this issue, two different groups tried an audacious experiment: using genome editing, they gradually merged a yeast’s 16 chromosomes down to just one giant molecule. And, unexpectedly, the yeast were mostly fine.

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CRISPR DNA editing may cause serious genetic damage, researchers warn

CRISPR-Cas9, the gene-editing tool that is currently the darling of biotech and many other fields, may not be quite as miraculous as early tests suggested. A new study finds that what scientists thought of as a scalpel may be more like a felling axe, causing damage hundreds of times what was previously observed.

Before anyone panics and checks out the window for mutated monstrosities, it should be said right away that this isn’t a nightmare scenario by any means: the tool can still be used in many ways safely, and the clinical repercussions of the damage are unexplored. But this unexpected limitation of a tool so widely applied will almost certainly put a chill on its use.

CRISPR, as a quick reminder, is basically a molecule that cleanly and reliably snips bases out of DNA strands paired with a molecule that hunts out a single sequence of bases. Together, they act like a pair of laser-guided scissors.

The idea is that by cutting out a handful of bases in a sequence that produces, for instance, sickle cell anemia, you can disable that gene altogether. This has been shown in numerous studies, and although unexpected insertions and deletions (abbreviated “indels”) of a handful of base pairs has been observed, no greater damage has been expected or seen — until now.

It turns out that some CRISPR edits may produce indels at the scale of thousands of bases — more than enough to affect adjacent genes or otherwise interfere with normal genetic operation.

The study published today in Nature, by Michael Kosicki, Kärt Tomberg and Allan Bradley of the Wellcome Sanger Institute, explains that previous research may have never encountered this type of damage simply because, essentially, it never allowed damage at this scale to occur.

The problem isn’t that CRISPR is going wild and producing this damage on its own; instead, the issue is an unexpectedly sloppy repair job by the cell itself.

After a CRISPR snip, lead author Bradley explained in a Nature news writeup, “the cell will try to stitch things back together. But it doesn’t really know what bits of DNA lie adjacent to each other.”

While doing its best to repair the damage with a bit of its own genetic cutting and pasting, it may accidentally substitute hundreds or thousands of base pairs that weren’t there, or cut out similarly sized ranges that were supposed to remain.

Because previous studies often used many copies of the same thousand-pair (or thereabouts) sequence to watch CRISPR in action, the possibility of thousand-pair damage was pretty much absent. It’s only when using much longer and more diverse strands of DNA that these high-volume indels are possible.

“We speculate that current assessments may have missed a substantial proportion of potential genotypes generated by on-target Cas9 cutting and repair, some of which may have potential pathogenic consequences,” reads the paper.

Fortunately, the damage seems to only occur when the job performed by the CRISPR complex is the cutting out of a sequence, leaving it open for the cell to repair. There are other methods that involve replacing or deactivating sequences that should not provoke this reaction. And like many problems in the practical biological sciences, it doesn’t need to be feared and worried about — it needs to be studied and accounted for.

All the same, having serious genetic damage accompany any part of this revolutionary technique will surely (or at least hopefully) spur inquiry and countermeasures, even if it means tapping the brakes on certain existing therapies, experiments and companies.

Researchers edit coral genes, hope to understand how to save them

Coral reefs are the poster-organisms for ecosystem services, aiding fisheries, promoting biodiversity, and protecting land from heavy waves. Unfortunately, we seem to be repaying them by killing them. Our warming oceans are causing coral bleaching and death, rising sea levels will force them to move, and the acidification of our oceans will make it harder for them to form reefs. It would be nice if we could help them, but interventions are difficult to design when you don’t know enough about coral biology.

Now scientists have announced a new tool is available to study corals: genetic editing provided by the CRISPR/Cas9 system. The ability to selectively eliminate genes could help us understand how corals function normally and could eventually provide a tool that lets us help them ride out climate change.

Coral complexities

You might think that we’d have a pretty good grasp of coral biology, given the amount of study that reefs receive. But much of that study has focused on coral reefs as an ecosystem, rather than coral as an organism. And that’s a big barrier to helping these reef-builders survive in our changing world. To give one example, coral bleaching is caused by a heat-driven breakdown in the symbiosis between coral and a photosynthetic algae that provides the coral with food. Corals that live in warmer waters are clearly able to form partnerships with heat-tolerant algae, but the precise mechanics of which species partner with what algae aren’t well understood.

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