Are we ready for a gene-editing future?

Andrea Macdonald, founder of ideaXme sat down with molecular biologist Ellen Jorgensen. A synopsis of that interview is presented here.

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Ellen Jorgensen, molecular biologist and president of Biotech Without Borders (Source: Flickr)

ideaXme had the honor to speak with Dr. Ellen Jorgensen, a molecular biologist, co-founder and president of Biotech Without Borders. She is a strong advocate of involving citizens in scientific endeavors and enlightened us about the advances in the field of biotechnology. During the interview, she talked about the promising results biotechnology has to offer to mankind.

Jorgensen strongly believes in the power of CRISPR techniques to transform the world for the better. It has enabled scientists to grow healthy food, cure diseases and develop new medicines, all by manipulating sequences in DNA – the blueprint of life itself.

Advances in cancer treatment

Dr. Ellen Jorgensen talked about how biotechnology is employed in the cancer treatment called Immunotherapy using CRISPR.

Traditional cancer treatments like chemotherapy not only destroy the cancer cells but affect the healthy cells as well. One of the biggest hurdles in treating cancer is the ability of cancer cells to hide from the body’s natural defense mechanism i.e. the immune system. This procedure boosts the cells of the immune system to fight the cancerous cells only. CRISPR is a genome editing technique used to alter or delete the specific sequences of DNA of living organisms. Jorgensen likes to think of it as a ‘guided missile’.

‘It’s a two-part system made from proteins which a lot of biological technology relies on. In this case, it is a combination of protein and a nucleic acid called RNA, a close chemical cousin to DNA. And the beauty of it is the protein never changes. It’s pretty much the same whatever species you put into, or whatever editing you want to do. It doesn’t change. The way it works is the targeting mechanism targets this protein to a specific place in your DNA. It makes a cut through both strands of the DNA. That’s a catastrophic event for a cell.

So, it sends in its natural repair processes. You can hijack those processes to get them to do what you want. It’s a combination of something external and something internal. And so far, it’s been used mostly in cells that you can take out of the body and put back like the cells I was talking about. The immune system cells are reprogrammed to fight the cancer. CRISPR is being used to reprogram them. So, that’s an example of its biomedical use. It’s also being used in agricultural tech to more efficiently produce genetically modified plants and animals that have valuable characteristics like drought resistance.’

Progress in genome editing

‘Another controversial area is editing an entire species. Gene drive is like a perpetual motion machine for genome editing’, says Jorgensen.

Gene Drive relies on the use of CRISPR techniques to genetically modify organisms

‘One individual organism can spread a genetic characteristic through a population of the same species extremely quickly. So, it overrides normal Mendelian inheritance and all its progeny no matter what organism it mates with, whether they have the gene or not. All the progeny will carry the new edit. With a lethal edit you can make an organism less able to produce. Within 10 generations you can completely change over a species, even eliminate it. Work is being carried out with the mosquito strain that carries malaria in Zika in this area.’

Gene drive relies on the use of CRISPR techniques to genetically modify organisms. This technology helped control the spread of diseases like malaria through mosquitoes. Scientists targeted the specific gene responsible for the production of female mosquitoes and hence, suppressed their population by altering the genetic material in the chromosomes. The other technique was to use the CRISPR gene drive to modify the genes in the mosquitoes and make them resistant to malaria.

Democratizing science

Keeping people aware of the new things happening in the world is extremely important. With the research accessible by just the privileged beings like scientists, the general public is left fallen in the cracks.

According to Dr. Ellen Jorgensen, ‘If you don’t understand a technology and it is imposed on you; it is frightening. The process of doing some of the science yourself, even at a low level, has the potential to demystify it. So, at Biotech Without Borders we invite the public to do some of the science themselves.

It is a standalone laboratory, not part of a university or a company. It’s a non-profit entity that exists to serve the public. One of the things we do is to provide lab space for people who want to do projects and who may not otherwise have access to a lab. The other important part of the mission is outreach in the form of hosting all sorts of lectures and public forums around the technology and getting people involved in it ‘hands on’ within classes and workshops.’

Challenges faced in CRISPR techniques

Dr. Ellen Jorgensen shares the sentiment that gene editing systems are extremely challenging: Right now the bottle neck for most therapy with CRISPR, is delivery. If a person has a disease like cystic fibrosis, a disease for which we know the cause, we know exactly what letter in the DNA we need to change. It would be great if we could go into that person’s lungs and change it over to a more normal lung. But the delivery is the problem. You have to get inside the cells.

The hurdle for gene therapy and a lot of other advances is finding a way to target the right cells. How do you get the system inside the cells? And right now, we’re using technology that we’ve used for many years in the past, so you can deliver it with the virus. You can deliver it as a Nano-particle, but nobody really knows how efficient those deliveries are. There’s also a problem with potential ‘off target’ effects, where in a small proportion of cases, it edits what it is not supposed to edit. The edit produces something catastrophic, produces a cancer cell. That is of course bad.’

The ethical concerns

Although unexpected dangers of genome engineering do exist, they can, however, be minimized by introducing some regulations, ethical guidelines and a proper code of conduct to handle CRISPR experiments. Moreover, some people are concerned that it might pose serious threats to the community. Will CRISPR somehow end the genetic diversity organisms own? What if an experiment goes wrong on a human and turns it into a Frankenstein monster? These are some of the serious questions under consideration.

What if an experiment goes wrong on a human and turns it into a Frankenstein monster?

‘The huge question is Who regulates the stuff. Who should be given the remit to regulate it?” I don’t have a ready answer for that but it’s something that’s up in the air right now. If you want the public to ‘weigh in’ they have to know that it exists. If you ask the average person on the street, unless they are the type of person who is glued to the Discovery Channel or similar, they are not necessarily going to know what was regarded as science fiction for generations is now close to happening. One of my missions in life is to spread the word how fast the science is moving. I tell people that they should form their opinions of it.’

Jorgensen says, ‘It’s always challenging. If a technology is sufficiently powerful it’s always going to be dual use. You have to take a deep breath and do the best you can. I applaud DNA synthesis companies for not just sending out any sequence that anyone asks for. I applaud that there’s a certain amount of gate-keeping that they’re not going to send someone genes for ricin or small pox without very good reason. But at the same time, I don’t think that it should be heavily regulated.’

Procedures like genome-editing, cell programming, and genetically engineered food have the potential to offer solutions to the problems faced by humanity. With the rise of the Fourth Industrial Revolution, it has become a need of time to adapt to these new technologies but in a cautious way. So, are we ready for a CRISPR future?

With permission of ideaXme, a global podcast, ambassador and mentor programme. ideaXme interviews the creators and innovators who shape our world. They speak to all those who Move the human story forward! ideaXme Ltd.

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The original interview has been condensed and concluded by Iqra Naveed.

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