Gene Editing: Disrupting Today’s Therapeutics Industry And Revolutionizing The Future of Medicine http://ift.tt/2x57GsK


Gene Editing: Disrupting Today’s Therapeutics Industry And Revolutionizing The Future of Medicine

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Dr. André Choulika, CEO of gene editing company Cellectis, discusses how gene editing presents great promise for the future of medicine

What is gene editing? Gene editing is the ability to edit the DNA molecule or chromosomes that are inside of a living cell. It is similar to editing a text using a word processor to correct, insert or delete characters – but at the level of the DNA molecule itself inside of the living cell. Current technologies have made significant progress in the precision and power to edit the genes of any species, including humans.

Two years ago, the first patients were treated by gene edited CAR T-cells to cure severe cases of Leukemia. CAR Ts are T-cells for the human immune system that are genetically modified with a gene coding for a Chimeric Antigen Receptor, or CAR. The CAR redirects the killing properties of these T-cells against a patient’s cancer cells.

Through CAR T-cells, gene editing is already used in clinics today for some cancer therapies. Therapeutic gene editing provides a new way of treating patients by correcting the genetic roots of their diseases, rather than merely addressing the symptoms, opening the door to the treatment of patients who are born with severe genetic disorders.

Gene editing presents great promise for the future of medicine, even if we still need to be very cautious about what this future will bring. Remedying errors in the genome of patients in the same way that a programmer fixes bugs in a software program is an enticing idea, but it raises a series of ethical questions that are related to who we are and where we are headed.

Should, for example, the reproductive cells of patients be modified so that disadvantageous mutations are not transmitted to ensuing generations? What would the human germ line look like in a hundred years if gene editing and human therapy were limited to non-reproductive cells only, where tissues that are not part of the germ line or embryonic cells are treated? How would we determine if something should or should not be fixed? For example, should gene editing be used to improve IQ if a deficiency in IQ is found to be a handicap? Such questions could be extended to almost any and all of our “perceived” traits.

We can now imagine a day when the notion of perfecting the human genome is a real possibility. A fundamental question remains: what is a perfect human genome? If we cannot address this question today, we must at least ask ourselves the following:

  • Should we establish rules? Why now? Whose rules? According to whose ethics, religion, politics and interests?
  • Should we continue as we are? What kind of world is this leading us to? What new kinds of species, plants, animals and humans will live in the future?

We are already walking, not running, down this path. And it is a familiar path, for we have walked it these last 11,000 years, perhaps without even knowing it.

Today, a global consortium called the Human Genome Project-Write is synthesizing a full human chromosome of 246 million base pairs, chromosome 1. HGP-Write aims to synthesize the entire human genome from its constituent components.

Today, scientists are still at the stage of copyists and, by the way, whose genome are these scientists copying? Is it the genome of a real human or that of an invented one?

How will geneticists work 20 years from now? Geneticists will work according to the following five fundamental pillars of genetic science:

Genomics: Sequence everything that’s living on Earth. The entire genome of every living species will be deciphered, stored and made available to the public. At some point, all humans will have their genomes sequenced.

  1. Artificial Intelligence: Annotate genes and the way that metabolic pathways interact in an organism, from the simplest forms to the more complex ones and up to full organisms.

III. Gene Editing: Enable the direct intervention on genomes in living organisms to tune the program, add new features and make genome system “updates.”

  1. DNA Synthesis: DNA printers will be able to print DNA by the megabase (bases are the four chemical molecules that compose DNA: A, C, G and T), then by the gigabase and assemble them into chromosomes.
  2. Robotics: Unmanned experiments and procedures are performed by the thousands, then millions or more a day.

All of these technologies are already developing rapidly and making spectacular progress. Twenty years from now, biology labs will look more like the trading floors of a bank, with scientists sitting behind computer screens, rather than the labs that we know today. Experiments will happen on fully automated floors full of robotics.

What would a scientist do on these computers? They will practice synthetic biology and assemble metabolic pathways – simple ones at first, followed by more complex ones. Then, with the help of artificial intelligence and machine learning, they will start to assemble the pathways of complete organisms. Once a new organism is assembled and functionalities are tested, it will be translated into DNA that’s arranged in chromosomes, then sent to a DNA printer. The DNA printer will print the DNA molecule, assemble the chromosomes and the newly synthesized genome will be injected into emptied eggs to bring new life.

From copyists, humans will become authors – genome authors – creating life.

No one really knows what we will do to life on Earth, ours included, during the 21st century. However, we must ponder the questions that are posed by the staggering advances of the science of genetics. It is the responsibility of all, including scientists, medical practitioners, healers, politicians, lawyers, religious leaders and philosophers, to start this conversation in an effort to tackle the issue at hand.

Dr. André Choulika is the chairman and CEO of Cellectis. He received his Ph.D. in molecular virology from the University of Paris VI (Pierre et Marie Curie) and completed a research fellowship in the Harvard Medical School Department of Genetics. Later, while working in the Division of Molecular Medicine at Boston Children’s Hospital, Dr. Choulika developed the first approaches to meganuclease-based human gene therapy. 

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