Susana Machado • September 7, 2017

Chances are, you’ve already heard about the groundbreaking gene editing technology known as CRISPR. The technique has been in the headlines a lot lately due to its controversial function and plethora of research opportunities it presents. The prospective uses for CRISPR have captured the attention of the scientific community and general public. Here’s what you should know about CRISPR and its progress so far.

The Background

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. The technology was discovered as a foundational part of bacterial organism’s immune system and modified to be used for genomic engineering in 2013. Generally, when someone refers to CRISPR, they are likely talking about CRISPR-Cas9, where Cas9 is the protein that does the actual “snipping” of the DNA.

 

Since its identification, CRISPR has faced quite a few controversies, including speculation that use of the technique would lead to “designer babies,” or parents selecting for specific characteristics like hair or eye color.

 

That future won’t come fast however, as the FDA has kept a watchful eye on CRISPR and the research projects it chooses to approve using the tech. Currently, CRISPR-edited embryos are only allowed to mature for a few days in the U.S., though more ambitious research with embryos has taken place in China.

The Potential

Armed with the power of gene editing, there’s a lot you can do. Though the technique was only discovered a few years ago, several research products have been conducted with drastically different goals, but equally exciting results, here are just a few:

  • Removing a viral gene in pigs – At the beginning of August, Science published a study detailing the successful use of CRISPR to remove PERVs (genes that contain dangerous viral infections) from piglets. This success is a step toward xenotransplantation, or the transfer of organs across species. Pig organs are similar in size to human organs and parts of pig organs are already used in some surgeries like valve replacement. Currently, about 22 people die daily waiting for an organ transplant. Xenotransplantation still has a ways to go, but CRISPR presents a promising solution to the problems it has faced in the past.
  • More accurate mutation removal – Researchers in Oregon recently conducted an experiment to remove a genetic mutation that affects the heart and the results are promising. Chinese scientists conducted a similar study in 2015, but CRISPR was found to have unintended effects on the genome, such as removal or mutations of genes it was meant not to target. The Oregon scientists seems to have limited that effect as according to their release, “there were no off-target mutations.”
  • Storage space … in your genes – Back in July, Harvard researchers published a paper about storing a gif of a galloping horse in bacterial DNA using CRISPR. The study was largely a proof of concept as the researchers end goal is to code cells to take in information about themselves and store it in the genome for scientists to review. The researchers are most interested in gathering information from brain cells to understand the process by which they become a neuron.
  • No more sickle cell – The ability to edit genes means the ability to eradicate genetic disease altogether. Since sickle cell is caused by a single mutation, it makes an optimal candidate for the use CRISPR.  U.S. clinical trials have yet to begin, but the NIH is set to launch a study later this month.

There’s plenty of other research on CRISPR going on right now and more studies are coming. What do you think about the use of this technology? Are scientists being cautious enough about how and what they use CRISPR for? Where do you see the future of gene editing going? Let us know on social media by using #angelMDCRISPR in your post.

Further reading

The Gene: An Intimate History by Siddhartha Mukherjee – If you’re as interested in the genome and genetics as I am, then this is a must-read. Mukherjee chronicles the entire exciting, and at times frightening, history of genetic research from Mendel’s pea plants to a tragic death as a result of gene therapy. Mukherjee interweaves his own family’s genetic history with schizophrenia in an enlightening explanation of the impacts of genetic research.

A Crack in Creation: Gene Editing and the Unthinkable Power to Control Evolution by Jennifer Doudna and Samuel Sternberg – If Doudna’s name looks familiar to you, it’s because she is one of the scientists who first discovered and popularized CRISPR. In her new book (which I am anxiously awaiting to come off hold at the library), she grapples with the consequences of her discovery, something she also discussed with Wired earlier in the summer.

Epigenetics: How Environment Shapes our Genes by Richard Francis – First published in 2011, there are now multiple editions of this dive into a fascinating subsection of genetics. Francis explains the different aspects of epigenetics, including how the idea came to be and how it can be observed throughout history and today.

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