Gene-editing technology may cure deadly diseases

September 13, 2017
By Thom Wellington

Even though CRISPR sounds like something as simple as a cereal or a toaster treat, it is actually a scientific development that is making research faster and cheaper with more accurate results.

CRISPR is a powerful gene-editing technology that is already showing promise in curing difficult and deadly diseases. CRISPR is a defense mechanism found in a wide range of bacteria that was first accidently identified as changes in the DNA sequence by Japanese scientists. Later, research by Francisco Mojica at the University of Alicante noted the unusual DNA structure was linked to an invading virus. Interest continued to grow around the world as researchers investigated surviving bacteria which had one property in common: they all contained CRISPR molecules to create a natural defense system.

This unusual pattern in some bacterial genomes, a repeated sequence of DNA, is now known as “clustered regularly interspaced short palindromic repeats” – CRISPR. Once researchers realized the unique repeating sequences matched the DNA of viruses everything changed and the doors began to open for a different approach to cures.

What makes CRISPR worth the attention is that this new tool allows medical scientists the ability to edit genomes with exact precision. CRISPR cuts years off studies and provides exacting efficiency. CRISPR is far better and more reliable than older techniques for gene splicing and editing. According to Randall Platt, part of Feng Zhang’s research team at the Broad Institute of Harvard and MIT, when discussing the advantages of using CRISPR to follow cancer mutations in cells, “in the past, this would have taken the field a decade, and would have required a consortium. With CRISPR, it took me four months to do it myself.” This efficient, yet much faster, method of research translates into a wide range of studies underway around the world.

Recent experiments by Zhang’s team at the Broad Institute, made possible by CRISPR technology, include work on brain function genetic mutations linked to autism and schizophrenia. Other related research includes targeting AIDS, Lyme disease, cataracts and Clostridium difficile.

Basically, researchers can program DNA with instructions for modified RNA that can target an exact antibiotic-resistance gene.

“Once the phage infects a bacterium, the guide RNA latches on to the resistance gene. That prompts an enzyme called Cas3, which the bacterium normally produces to kill phages, to destroy that genetic sequence instead,” according to Sara Reardon in Nature. The Cas3 eventually destroys all of the DNA, killing the bacterium.

In simple terms, CRISPR is one part of the bacteria’s immune system, which stores fragments of bad viruses so it can later recognize and defend itself the next time that virus attacks. The research is already showing promise in combating antibiotic-resistant infections along with cancer and other major diseases. Early tests have shown laboratory mice surviving antibiotic-resistant infections that would have otherwise killed them. The mouse genome is similar to a human’s, but mice reproduce every three weeks, which allows scientists to monitor several generations in a short period of time.

This innovative technique for cutting and pasting genes has already created revolutionary results this year alone, including:
• Scientists have successfully removed HIV from a living organism using CRISPR, and removed both acute and latent infections.
• CRISPR was successfully used to target genes that can trigger abnormal tumor growth, and actually shrunk prostate and liver cancer cells in mice.
• Targeting the protein called Tudor-SN with CRISPR shows promise in inhibiting fast-growing cancer cells.
• Researchers have now been able to modify the genes to protect themselves from superbugs. This alone shows promise with multidrug-resistant organisms that could be cured with modifying DNA.

These early successes due to CRISPR-involved research have caught the attention of venture capital firms. They are investing millions of dollars at research centers across the world, attempting to be in on the next medical breakthrough. To secure the research, patent filings have exponentially increased. Patent holders would have the right to licensing fees, which may be worth a fortune, and promising innovation seems likely in many studies.

Thom Wellington
Will the innovations create other problems with changes to a DNA structure? The movie “Jurassic Park” offered a fictional glimpse at what can happen with DNA transformations. You may recall the cloning of a lamb called Dolly in 1996 by Scottish scientists. Luckily, researchers are currently only focused on mice, and say they can easily turn off a DNA change that gets out of control now that they have a tool that can program immediate changes rather than waiting for generations of mutations.

Everyone wants to see the advancement of science and medicine that benefits mankind. Research enhanced by tools such as CRISPR are moving us closer to medical innovation from internal DNA tweaking rather than solely relying on drug solutions. As humans, we carry genes that may protect us from one disease, but increase our susceptibility to others. Combining the advancements in pharmaceutical research with innovative biological engineering could produce cures to some of our worst ailments and diseases. CRISPR may be something we remember as the keystone in making miracle medical science.

About the author: Thom Wellington is the CEO and a stockholder in Infection Control University.