A Gene Editing Breakthrough, Opening the doors for new developments

A Gene Editing Breakthrough, Opening the doors for new developments

“There is still something in everything I do that defeats me, makes me dissatisfied, challenges me to further effort. Sometimes I rise above my level, sometimes I fall below it, but always I fall short of the things I dream.”
― H.G. Wells, The Island of Dr. Moreau

By: Sarfraz Nawaz

Are you feeling a little more lethargic as you get older? Let’s just tweak your genome a bit by inserting a slice of leopard DNA. Done! Want better eyesight? A bit of eagle eye blended into your genome and you’re good to go. This may sound farfetched, but it is now a reality with the remarkable feat of genome editing achieved in the United States recently.

In 1896, a prolific English writer and novelist H.G. Wells published his science fiction novel “The Island of Dr. Moreau”. The novel spurs the reader to consider the limits of natural science and the distinction between men and beasts. Its plot focuses on a shipwrecked English sailor, an upper class gentleman named Prendick, finding himself shipwrecked in the ocean. He is rescued and taken to a remote island in the South Pacific. The island’s inhabitants are animal-human hybrids. They are created through painful surgical procedures by Doctor Moreau, who is trying to perfect the process of transforming animals into humans. The hybrids inevitably revert to animal form. The sailor escapes the island, but not before the hybrids return to the jungle and the Doctor’s compound burns to the ground.

The news that ‘for the very first time in history, researchers in the United States have used gene editing in human embryos’ came as a reminiscent of that aptly-built plot of the novel.

Newspapers around the world were full of praise for the successful embryo editing by the research team led by Oregon Health and Science University. Reports suggested that the team of the US scientists used “genetic scissors,” called CRISPR-Cas9, to target and remove a mutation associated with hypertrophic cardiomyopathy, a common inherited heart disease, in 42 embryos. The success in this experiments means that, now humans are, for the first time, able to edit (add, delete, or change) their own genome – a remarkable achievement, indeed.

The successful modification of human embryos to rectify a genetic flaw holds plenty of hope for families suffering from hereditary diseases. Scientists who want to explore the technique hail it as a biomedical advance that could one day give people the option not to pass down heritable diseases. The tool could also reduce the number of embryos that are discarded during fertility treatments because of worrisome genetic mutations.

Technically, this development is an incredible scientific advancement. CRISPR-Cas9, the tool used for genome editing, has proven to be a very efficient mechanism to perform complex genetic manipulations.

The potential medical benefits from genome editing are significant. We might be able to edit a foetus’ DNA to remove inherited defects before birth. We might also be able to craft personalized medical procedures that are tailored to each person’s DNA, which could help alleviate dangerous side effects from pharmaceuticals while increasing the drugs’ efficacy. We might even be able to increase longevity by slowing down or even halting the ageing process through gene therapy.

The ethical implications of genome editing are equally significant. Performing experiments on human embryos is already the subject of much debate. These new procedures have the potential to more dramatically alter living tissue, which could lead to unintended moral dilemmas.

Altering genes in sperm, eggs or embryos can spread those changes to future generations, so-called “germline” engineering. But it’s ethically charged because future generations couldn’t consent, any long-term negative effects might not become apparent for years, and there’s concern about babies designed with enhanced traits rather than to prevent disease. Critics counter that it takes more than safety or even efficacy to make a procedure ethical.

“The scientists are out of control,” says George Annas, director of the Center for Health Law, Ethics & Human Rights at the Boston University School of Public Health, who thinks that scientists should not edit the genomes of human embryos for any reason. “They want to control nature, but they can’t control themselves.”

If genome editing becomes commonplace, couples could procure designer babies, with all the physical traits they desire. Blue eyes? Lighter skin tone? Increased muscle mass? No problem. But what would stop some rogue clinic from becoming the modern-day Island of Doctor Moreau?

Most pundits assumed humans would be augmented with mechanical aids to improve performance, including brain capabilities. In the genome-editing model, we’re changing biologically instead. Like rapid advances in artificial intelligence, researchers may have opened a Pandora’s Box that could lead us to places we can’t begin to understand.

What is Gene Editing?

While scientists have long been able to find defective genes, fixing them has been so cumbersome that it has slowed development of genetic therapies. There are several gene-editing methods, but a tool called CRISPR-Cas9 has sparked a boom in research as laboratories worldwide adopted it over the past five years because it’s faster, cheaper, simple to use with minimal training and allows manipulation of multiple genes at the same time.

How It Works?

Pieces of RNA are engineered to be a guide that homes in on the targeted stretch of genetic material. The Cas9 is an enzyme that acts like molecular scissors to snip that spot. That allows scientists to delete, repair or replace a particular gene

Leave a Reply

Your email address will not be published.