Wellness

BEAUTIFUL BREAKTHROUGH CURES: CRISPR

by John Liebler, founder of Art of the Cell. “CRISPR Cas9 System.”

CRISPR

For the first time, scientists have the ability to alter the DNA of every living organism on the planet -- including humans! They can cure diseases and improve life in ways never before possible. And, they can do it beautifully, with speed and precision.

In the past 5 years, the new gene-editing technology known as CRISPR, has transformed biology. Researchers around the world are now using CRISPR, an acronym for Clustered

Regularly Interspaced Short Palindromic Repeats, to cure cancer and other diseases, as well as correct major genetic flaws, including the gene mutations responsible for muscular dystrophy, cystic fibrosis, hepatitis, and HIV.

Other groundbreaking applications for CRISPR technology have made major advances in everything from organ transplants to developing crops that are naturally resistant to pests.

CRISPR is widely considered by far to be the biggest scientific breakthrough of the century.

It is generating much excitement and promise, and has resulted in one of the highest-stakes races in the biopharmaceutical industry. Labs around the world are each striving to be the first with approved CRISPR-based treatments.

CRISPR genome editing illustration showing DNA helix.

The first CRISPR-based trial was conducted by a team lead by Lu You, at Sichuan University, Chengdu, China, in October 2016. They tested cells modified using the CRISPR–Cas9 gene-editing technique in people with lung cancer. Now, dozens of CRISPR-based trials are planned worldwide and China is leading the pack, with the U.S. trying to catch up.

CRISPR gene editing tool shown with printout of gene map.

New Scientist recently reported that “as many as 20 human trials” of CRISPR would soon be launched, mainly in China. Currently, there are 9 CRISPR-based trials of listed on ClinicalTrials.gov.

Molecule model of CRISPR tool and E. coli DNA fragment.

After almost 30 years of research on gene editing, CRISPR hails as a simple elegant system. The system is natural to the bacterial immune system, helping them defend against the DNA of attacking viruses. It enables the programming of protein, using a single RNA, to cleave almost any DNA sequence. The latest permutation, CRISPR/Cas9, is often referred to as “programmable molecular scissors.”

CRISPR gene editing tool shown snipping strand of DNA.

CRISPR technology has already transformed plant and microbial research. Now it is doing the same with animal models.

Preclinical studies with CRISPR have shown that it can shrink human prostate cancer tumors in mouse xenografts and it can successfully treat mice with Duchenne–like muscular dystrophy. Studies of embryos, which have shown that CRISPR can help to repair their genes.

Gene editing method called CRISPR uses bacterial proteins to cut apart DNA.

CRISPR will be much more powerful than the antibodies in curing cancer and other diseases, according to Timothy Chan, who does clinical research in immunotherapy at Memorial Sloan Kettering Cancer Center in New York City

The possibilities are thrilling. They offer a lot of hope to advance healthcare, agriculture, and other life sciences. CRISPR places an entirely new kind of power into human hands. And although there are still technical challenges and ethical questions on the table, the race is on!

See more exciting stories about DNA in our post Gold Water Cocktails Save Lives.

CRISPR gene editing tool at work on a strand of DNA.

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CRISPR/Cas9 genome editing tool at work.

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Multi-colored model of CRISPR gene editing tool.

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Gene editing tool known as CRISPR shown snipping DNA strands.

IMAGE LIST

Image: by John Liebler, founder of Art of the Cell. “CRISPR Cas9 System.”
Image: by Feng Zhang. Courtesy of McGovern Institute for Brain Research/MIT. CRISPR-Cas9 method for genome editing, explained.
Image: “CRISPR in Neuroscience: How Precision Gene Editing May Unravel How the Brain Works (and Why it Sometimes Doesn't).” Courtesy of Harvard University.
Image: A molecule model of a CRISPR (clustered regularly interspaced short palindromic repeats) and a DNA fragment from E. coli. Courtesy of Wikimedia Commons.
Image: by Jennifer Doudna. UC Berkeley. CRISPR.
Image: by Val Altounian/Science. “A popular gene editing method called CRISPR uses bacterial proteins such as Cas9 and RNA to cut apart DNA.”
Image: Courtesy of World Science Festival. “Groundbreaking Discoveries to the Public – CRISPR: The Editing of Human Genes.”
Image: by Steve Dixon. “CRISPR/Cas9 genome editing tool at work.”
Image: Courtesy of NIH Image Gallery. “Cas9 from CRISPR.”
Image: Courtesy of National Human Genome Research Institute (NHGRI). “CRISPR Cas9.”