Host of genetic research groups issue backing for inheritable human gene editing

A group of 11 organisations spanning five continents have issued a policy statement in support of current efforts to edit human germline genomes – that is, genes which passed down from parent to child.

The statement, published today in The American Journal of Human Genetics, said that the organisations support publicly funded in-vitro research into germline genome editing, which could eventually be used to eliminate devastating inherited diseases.

However, they stopped short of supporting research that would lead to a human pregnancy, arguing that at this stage it would be inappropriate to undertake.

“Our workgroup on genome editing included experts in several subfields of human genetics as well as from countries with varying health systems and research infrastructure,” said statement lead author Kelly E Ormond, professor of genetics at Stanford University.

“Given this diversity of perspective, we are encouraged by the agreement we were able to reach and hope it speaks to the soundness and wider acceptability of our recommendations.”

A depiction of the now widespread CRISPR-CAS9 gene editing complex, which enables DNA to be edited. Image courtesy of Image courtesy of Ian Slaymaker, Broad Institute

Germline genome editing has only been even hypothetically possible very recently, with the development of the CRISPR/Cas9 genome editing tool in 2013. Allowing precise customisation of genes, it creates whole new possibilities for genetic research, particularly in humans.

However, while scientists feel confident of the technological and scientific prospects, the technology raises significant ethical issues, particularly in germline genomes, where any changes could have an impact for generations.

As a result, the scientists argue that there needs to be strong public discussion about the ethical issues, as well as clear rationale, evidence and ethical justifications for any future research in the area.

“While germline genome editing could theoretically be used to prevent a child being born with a genetic disease, its potential use also raises a multitude of scientific, ethical, and policy questions,” said Derek T Scholes, ASHG director of science policy. “These questions cannot all be answered by scientists alone, but also need to be debated by society.”

“As basic science research into genome editing progresses in the coming years, we urge stakeholders to have these important ethical and social discussions in tandem,” added Ormond.

Conventional two-stranded DNA

As a result, it is likely that we will see far more public discussion before the first gene-edited babies come anywhere close to being conceived.

The statement was authored by the American Society of Human Genetics (ASHG), the Association of Genetic Nurses and Counsellors, the Canadian Association of Genetic Counsellors, the International Genetic Epidemiology Society, and the National Society of Genetic Counselors. It was also endorsed by the American Society for Reproductive Medicine, the Asia Pacific Society of Human Genetics, the British Society for Genetic Medicine, the Human Genetics Society of Australasia, the Professional Society of Genetic Counselors in Asia and the Southern African Society for Human Genetics.

Steve “Woz” Wozniak to advise hologram emoji company that he calls “groundbreaking”

Apple’s co-founder Steve “Woz” Wozniak has found himself a new gig; Woz has joined the hologram emoji company, Mojiit, as an adviser.

In his role as advisor to Mojiit, the legendary entrepreneur and engineer will help assemble a world-class engineering team in addition to bringing investors and partnerships to the newly launched startup. Wozniak will also serve as mentor to Mojiit founder, Jeremy Greene.

“I’m thrilled to join Mojiit as an advisor,” said Wozniak. “Jeremy is a natural leader, the company is groundbreaking, it’s going to change the ecommerce space, and it’s a lot of fun.”

Created in 2017, Mojiit is the latest startup technology venture from Greene. The company’s tech essentially enables users to project and share 3D hologram emojis via smartphones.

The platform turns users into emojis by scanning their face, which can then be sent to loved ones and friends. Once a Mojiit message is received, it will map the area where it is received and place the Mojiit hologram there in real time, so it works in a similar way to Pokemon Go.

“Steve is one of the best and brilliant engineers in the entire world. But outside of that, he’s a wonderful man,” said Greene. “There isn’t anyone I’d want to be in business with more than this guy. He’s a legend. Who better to learn from than the guy who created the computer?”

Image courtesy of Nichollas Harrison. Featured image courtesy of Mojiit

In addition to consumer use, businesses of all kinds can tap into hologram emojis with Mojiit’s technology.

Mojiit investors already  include NFL alum Ed Reed, and the company was able to raise a total of $1 million in its seed round of funding.

Alongside the appointment of Woz, Entourage and Ballers producer Rob Weiss recently joined the company as a creative director.

“It’s exciting to expand beyond television and film to digital platforms,” said Weiss. “Hologram technology brings incredible opportunity to entertainment and media. I’m thrilled to be leading creative at Mojiit.”

Nanoengineers send antibiotic-delivering micromotors into the body to treat cancer-causing infection

Nanoengineers have demonstrated for the first time how “micromotors” that measure half the width of a human hair can be used to transport antibiotics through the body.

Nanoengineers at the University of California San Diego tested the micromotors in mice with Helicobacter pylori infections, which can also be found in about two-thirds of the world’s population and while many people will never notice any signs of its presence it can cause peptic ulcers and stomach cancer.

The mice received the micromotors – packed with a clinical dose of the antibiotic clarithromycin – orally once a day for five consecutive days.

Afterwards, nanoengineers evaluated the bacterial count in each mouse stomach and found that treatment with the micromotors was slightly more effective than when the same dose of antibiotic was given in combination with proton pump inhibitors, which also suppress gastric acid production.

Micromotors administered to the mice swam rapidly throughout the stomach while neutralising gastric acid, which can be destructive to orally administered drugs such as antibiotics and protein-based pharmaceuticals.

Because gastric acid is so destructive to traditional antibiotics drugs used to treat bacterial infections, ulcers and other diseases in the stomach are normally taken with additional substances, called proton pump inhibitors.

But when taken over longer periods or in high doses, proton pump inhibitors can cause adverse side effects including headaches, diarrhea and fatigue. In more serious cases, they can cause anxiety or depression.

The micromotors, however, have a built-in mechanism that neutralises gastric acid and effectively deliver their drug payloads in the stomach without requiring the use of proton pump inhibitors.

“It’s a one-step treatment with these micromotors, combining acid neutralisation with therapeutic action,” said Berta Esteban-Fernández de Ávila, a postdoctoral scholar in Wang’s research group at UC San Diego and a co-first author of the paper.

The nanoengineers say that while the present results are promising, this work is still at an early stage.

To test their work, the team is planning future studies to into the therapeutic performance of the micromotors in animals and humans, and will compare it with other standard therapies used to combat stomach diseases.

UC San Diego nanoengineers also plan to test different drug combinations with the micromotors to treat multiple diseases in the stomach or in different sections of the gastrointestinal tract.

Overall, the researchers say that this work opens the door to the use of synthetic motors as active delivery platforms in the treatment of diseases.

Image and video courtesy of the Laboratory for Nanobioelectronics at UC San Diego.