A New Age In Functional Genomics !

We know that ,Gene therapy is the use of genetic material to modify or manipulate the expression of a gene product or in other words, to alter the biological properties of living cells for treatment use. Gene administration offer the potential to treat diseases for which no or few treatments exist.
Advances in gene editing technology have made it possible for amateurs to transform their genes outside the lab.

Josiah Zayner, 36, was a NASA biologist. Now, in order to gain muscle, he became the first person to genetically modify himself with the revolutionary gene editing tool CRISPR-Cas9. However, unlike biologists in the general sense, he did not complete the trial at hospitals, university labs, but his own garage and kitchen

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During the experiment, Josiah Zayner knocked out the Myostatin (MSTN) gene with CRISPR technology and hoped to strengthen her own muscle. Past studies suggest that the MSTN gene is highly expressed in skeletal muscle of the animal body and is an important gene that negatively regulates muscle growth. Many scientific studies have shown that the expression level of the gene is negatively correlated with the change of muscle weight, and its mutation will cause muscle hypertrophy.

In 2015, Chinese scientists successfully used CRISPR technology to successfully breed two MSTN knockout dogs. Knockout refers to the functional inactivation of the target gene. This method stops a specific gene in the animal from playing its role. In this study, the muscle of the knockout dog obtained was more developed at 4 months of age than ordinary dogs, and had greater athletic ability after adulthood. However, knocking out MSTN gene can cause the growth of human muscle, has not been confirmed.

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The results scientists have achieved in animal experiments add to the confidence of Josiah Zayner, who performed a muscle-building test on his left forearm. "Of course I do not like these dogs," said Josiah Zayner. "The DNA only gets into the area I'm injecting."

In a bio-hacking activity in August 2017, Josiah Zayner presented the creature hackers present with how to perform genetic engineering in the garage.

In an interview with Josiah Zayner in December 2017 by the British Guardian, Joshuah Zayner said when asked whether his arm is currently changing, four to six months later in a similar animal study will see results. "I hope some of the cells in my arm have changed, but I'm still developing some tests to detect the change."

There is not only Josiah Zayner who has the courage to carry out genetic engineering work on his own. Some people even hope that they will cure their own diseases by changing their own genes through DIY. In October 2017, Tristan Roberts, a 28-year-old software engineer who is also a member of the biological hacking community, ran live on Facebook on his gene therapy and sat calmly on a black leather couch in front of a glass table With needles and other tools, he hopes to use gene engineering methods to personally cure their own AIDS.

Six years ago, Roberts was diagnosed with AIDS. After two years of routine treatment, he said he "hated" the side effects of the drug and he hoped to cure AIDS by one of the other ways. He completed the injection in his abdomen, and the fluid he injected contained billions of plasmids, which are thought to promote the production of N6 antibodies. In 2016, the NIH research team found that under laboratory conditions, N6 antibodies neutralize 98% of HIV strains, a finding that is thought to be a new way to treat and prevent the spread of AIDS in the future.

Mechanisms of gene editing
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Official ban

Interest in bio-hacking has started to rise and cheap genetic material is becoming more readily available. Coupled with a tutorial on using CRISPR to edit genes on the Internet, U.S. officials and research organizations have voiced opposition. Recently, the U.S. Food and Drug Administration (FDA) and the American Society of Gene and Cell Therapy (ASGCT) issued separate statements on their websites.

On November 21 this year, the FDA issued a statement on its website that made it clear that any use of CRISPR-Cas9 gene editing in humans is considered gene therapy and that gene therapy products should be evaluated and researched by FDA's biologicals Center (CBER) to regulate. The FDA has realized that people now have easy access to gene therapy products for DIY, all of which are illegal, and the FDA is very concerned about the potential safety hazard.

Although the FDA does not mention the name of any company or product, Josiah Zayner believes the information the FDA publishes is for him. Because Josiah Zayner not only tested himself, but also owned a company that sells CRISPR DIY related products, a set of CRISPR / Cas9 tools knocking out the MSTN gene for just $ 20 on his company's website. In the definition of gene therapy by the FDA, even though the purpose of gene editing is not to treat the disease, it is still within the scope of gene therapy. As a result, CRISPR products sold by Josiah Zayner are also under the supervision of the authorities and selling without approval is illegal.

On December 7, 2017, ASGCT said in its statement that as the world's largest professional organization for gene and cell therapies, ASGCT does not support the definition of gene therapy as "gene editing using CRISPR in the human body." Regulated gene therapy, because of the potential risks of this process, and almost impossible to achieve therapeutic effect. ASGCT strongly opposes individuals for DIY gene therapy.

The best gene editing era?

CRISPR, which has the title of Gene Monster, is not the first gene editing tool in the world, but by far the easiest, cheapest, and most efficient gene editing tool. This is why creature hackers choose CRISPR for gene editing DIY , And sell cheap CRISPR-related biological products one of the reasons.

Before 2012, scientists had two major tools for gene editing. One was Zinc Finger Nurse (ZFN), and the other was TALEN (Transcription Activator-Like Effector Nuclease).

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Both use protein to calibrate the position of the DNA, followed by cleavage of the gene to be edited with Fokl nuclease, followed by insertion or removal. However, the proteins that ZFN and TALEN use to identify DNA are not easy to make, and ZFN is toxic to cells. TALEN is not easily introduced efficiently because of its large molecular mass, so scientists often have to spend a lot of time editing it A gene, or the work of gene editing to biological companies.

Now, in the lab, scientists can use CRISPR technology to simply and efficiently edit genes. "We can imagine that the old genetic engineering technology is like upgrading your PC every time you install new software, and CRISPR technology is like the genome software that uses these small pieces of RNA and we can simply edit It's, "said Jennifer Doudna, a professor at the University of California, Berkeley, one of the inventors of the CRISPR technology.

The CRISPR-Cas9 system has two major components, the Cas9 enzyme, which cleaves DNA like a "scissor" and the other is an RNA small molecule that directs "scissors" to a specific DNA sequence.

Since CRISPR - Cas9 is cheap and easy to operate, almost all laboratories can follow the instructions to conduct experiments. Since 2012, the number of relevant articles has increased exponentially. According to statistics, there are 277 scientific papers in 2013, 587 in 2014 and 2015 In more than a thousand papers.

At present, CRISPR greatly accelerated the progress of gene therapy. In 2016, the first human clinical trial to use CRISPR was launched. Several CRISPR technology-based start-ups around the world are also working toward clinical application. However, gene editing can also bring unexpected problems which may be visible after some more duration.

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• https://www.yourgenome.org/facts/what-is-a-gene
• https://www.theguardian.com/science/2017/dec/24/josiah-zayner-diy-gene-editing-therapy-crispr-intervie
• http://hplusmagazine.com/tag/gene-editing/
• https://www.cancer.gov/about-cancer/causes-prevention/research/crispr
• https://www.fda.gov/biologicsbloodvaccines/cellulargenetherapyproducts/ucm586343.htm

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