Kozubek - Modern Prometheus: Editing the Human Genome with Crispr-Cas9

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Jim Kozubek 2016, 2018

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Sections of this book previously appeared in STAT, The Atlantic , The Boston Globe , Scientific American, TIME and Nautilus .

Between 14 October 2013 and 6 May 2016, Jim Kozubek worked as a staff scientist at the Brigham and Womens Hospital which is affiliated to the Broad Institute of MIT and Harvard. Although the Broad Institute is in Crispr genome editing research, development, and sharing, this book was developed independently of the authors Broad affiliation.

This is a book about Crispr. It is animated by many forces. I grew up reading science books that could be described as instructive or didactic, but I saw the trade begin to gradually drift into a pop science which worsened an already existing problem that much of scientific explanation is based on partial truths or weak causal links. To break from this trend, I set out to write in a different style, which is the tragic vein of literature. To this end, the book puts an emphasis on scientists as fallible agents, and is injurious throughout, while taking few moral positions. It is not designed to attack or damage anyone per se, but to describe a more realistic, harder and more complicated situation which we endure.

Technology is accelerating. We have begun inserting ourselves into evolution, using the Crispr system to modify the genetic code of plants, sea creatures and livestock to reduce infection and promote the yields of crops. Crispr has been used to fix recessive conditions such as kidney disease in inbred Dalmatians, create super-strong beagles, cows without horns, miniature pet pigs, and it has been used to disable immune-alerting genes in pigs so that their organs can be used for human transplant. It is being used to alter the genes of mice to stop Lyme disease in the transmission cycle and to modify mosquitoes to stop the spread of Zika virus. Crispr is also being used in ways that are dubious. It has been used to disrupt genes in butterflies to affect color patterns in their wings, and as scientists suggest, it will soon be used to create customized butterflies with pretty new wing colors. Crispr is sold on the internet in kits, and is actively being used to do fiddling things, such as to create fluorescent beer. Its ubiquity and ease of use has also raised concerns about biohackers, who view gene modification as a right and alter microbes and organisms. But bio-terrorists might use it to turn common microbes into a pathogenic weapon. The US military started a program called Safe Genes to gene modify organisms to be used in battle and anti-Crispr tools to disable bio-weapons. Mail-Order Crispr Kits Allow Absolutely Anyone to Hack DNA, declared the headline of a November 2017 article in Scientific American . The iconoclast scientist Josiah Zayner has used Crispr to hack into his own genes.

Most controversially, Crispr is being used to modify human genes as a logical extension of what is called gene therapy, a decades-old strategy to slip a supplementary copy of a gene into a human cell by packaging it into a virus. Viruses can be engineered to work as tiny crafts to pilot bits of restorative gene code into our cells. Some of these viruses, such as adeno-associated virus, slip into our cells but dont integrate into a chromosome as a permanent fixture, while other viruses, such as gammaretrovirus and lentivirus, do install in a chromosome. In many ways, modern gene therapy is coming of age, effectively being used to modify the genes in the cells of living humans to treat eye diseases, which can cause blindness, such as Leber congenital amaurosis; promote the growth of healthy skin to treat the rare skin-blistering disease epidermolysis bullosa; or add supplementary copies of working genes that fix rare blood or immune system disorders, such as Severe Combined Immunodeficiency Disorder.