Co-Inventor of CRISPR Asking For “Global Pause”

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Late last year, Geneticist Jennifer Doudna, who co-invented a groundbreaking new technology for editing genes, called CRISPR-Cas9, gave a Ted Talk on the subject, entitled: “We Can Now Edit Our DNA. But Let’s Do it Wisely.” The implications for new parents are numerous.

In the video, she explains that CRISPR is actually an adaptive immune system (that is derived from the strep bacteria) that can be harnessed as a genetic engineering tool to potentially cure any genetic disease. Doudna also touches on the ethics in applying CRISPR to human beings.

For example, most women at risk of passing on faulty mitochondria to their newborns carry some healthy and some mutated mitochondria, In this case, CRISPR could be used to lower the number of mutated mitochondria in their eggs. Scientists say that “harmful effects would only kick in once the number of mutated mitochondria crosses a threshold, so this may be enough to prevent disease in their child, and perhaps in future generations too.”

Scientists are currently exploring CRISPR to:

1. Treat disease by modifying genes in adults (Human trials are now under way)

Pros: Could be used to treat all kinds of diseases and disorders
Cons: Could turn cells cancerous if the wrong bit of DNA gets modified

2. Prevent disease by destroying mutant mitochondria (Testing in human cells is about to start)

Pros: Could prevent mitochondrial diseases without using a donor
Cons: The effects are inheritable, so it’s ethically controversial

3. Prevent disease by modifying genes in embryos (Testing in human embryos is now under way)

Pros: Could prevent many genetic disorders, even before a baby is born, or alter his/her eye color or other characteristics
Cons: This is uncharted ethical territory; treatments are still a long way off

It should be noted that precise genome engineering has the potential to alter not just us, but the Earth itself. CRISPR could become a major force in ecology and conservation. It could, for example, be used to introduce genes that slowly kill off the mosquitoes spreading malaria, or genes that “put the brakes on” invasive species, like weeds.

Understanding CRISPR

CRISPR is a dramatic improvement over the sequencing and copying of genes that’s been going on since the1970s. CRISPR allows scientists to make precise edits to DNA strands – which could lead to treatments for many genetic diseases – but on the flip side, could also be used to create “designer humans” in the future. As far as back the 1980s, scientists observed a strange pattern in some bacterial genomes. One DNA sequence would be repeated over and over again, with unique sequences in between the repeats. They called this odd configuration “clustered regularly inter-spaced short palindromic repeats,” or CRISPR.

Here’s the scientific description of how CRISPR works:

“Scientists realized that the unique sequences in between the repeats matched the DNA of viruses – specifically viruses that prey on bacteria. It turns out CRISPR is one part of the bacteria’s immune system, which keeps bits of dangerous viruses around so it can recognize and defend against those viruses next time they attack. The second part of the defense mechanism is a set of enzymes called Cas (CRISPR-associated proteins), which can precisely snip DNA and eradicate invading viruses.”

Doudna says that the older process of working with genes was much like having to rewire your computer every time you need to run a piece of software. CRISPR is like having a software you can run every time. The CRISPR technology acts like a sentinel; it goes through the tissue, detecting broken DNA, and fixing it. Already, CRISPR is showing great signs of being able to treat the mutations that cause HIV, cystic fibrosis, sickle cell anemia, Huntington’s disease, and blood-related disorders.

Most science experiments are done on a limited set of model organisms: mice, rats, zebra fish, and fruit flies. That’s mostly because these are the organisms scientists have studied most closely and know how to manipulate genetically. In the United States, trials are now being conducted in mice (to change the color of their coat, for example) and monkeys in the United States (mainly to test how to deliver the technology into the cells.) It’s been reported that despite the controversy, at least one group in the United States and several more in China are currently working with human embryos.

For information on how CRISPR is being used to potentially cure HIV, read more here.

Taking The Time to Get It Right

Doudna and other scientists have called for a “global pause.” Aside from the necessary discussion about the the ethical implications of using CRISPR in humans, Doudna says that it’s also critical that scientists perfect how they will apply the CRISPR technology so that they can “control and limit any off-target or unintended effects of using this tool.” Based on current venture capital investments in start up firms looking to commercialize the CRISPIR technology, Doudna predicts that within ten years, there will be approved therapies for CRISPR for adults.

CRISPR stands at the precipice. It’s a scientific breakthrough of immense proportions that carries with it a huge responsibility to ensure that future generations will benefit from the technology – that the effects of its eventual adoption will be positive in all respects.

Other sources include: CNN and The New York Times

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