Lifeboat Foundation: Safeguarding Humanity
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Category: bioengineering

Move over, gene-editing proteins—there’s a smaller, cheaper, more specific genetic engineering tool on the block: DNAzymes—small DNA molecules that can function like protein enzymes.

Researchers at the University of Illinois Urbana-Champaign have developed a technique that, for the first time, allows DNAzymes to target and cut double-stranded DNA, overcoming a significant limitation of the technology. DNAzymes have been used in biosensing, DNA computing and many other applications. However, when it comes to genetic engineering applications such as gene editing or , they have faced a challenge: DNAzymes have only been able to target sites on single-stranded DNA, while the DNA coding for genes in cells is double-stranded. The researchers published their new technique in the Journal of the American Chemical Society.

“DNAzymes have many advantages, including higher stability, smaller size and lower cost than protein enzymes. These advantages perfectly fit the requirement for genetic engineering tools,” said study leader Yi Lu, a professor of chemistry at Illinois. “No DNAzymes could alter double-stranded DNA until this work. By making that happen, we open the door for DNAzymes to enter the entire world of genetic engineering.”

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Quick, accurate and easy-to-use, CRISPR-Cas9 has made genomic editing more efficient—but at the same time has made human germline editing much more feasible, erasing many of the ethical barriers erected to prevent scientists from editing the genes of heredity.

“The ethical debate about what is now called human gene editing has gone on for more than 50 years,” writes Dr. John H. Evans, co-director of the Institute for Practical Ethics at the University of California, San Diego. “For nearly that entire time, there has been consensus that a moral divide exists between somatic and human germline editing.”

In an essay published in the Proceedings of the National Academy of Sciences (PNAS), Evans contends that many of the potent bioethical arguments that once made germline editing a verboten concept, have begun to dissolve in the era of CRISPR.

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STANFORD, Calif. — A groundbreaking “superhero” vaccine inspired by the DNA code of Olympic athletes could help transform society over the next decade, a top genetic scientist claims.

The vaccine would provide lifelong protection against three of the top ten leading causes of death, according to Euan Ashley, professor of medicine and genetics at Stanford University. The so-called “superhero” jab could offer simultaneous, long-term protection against heart disease, stroke, Alzheimer’s disease, and liver disease, thanks to advances in genetic engineering.

This breakthrough treatment would deliver the blueprint of “ideal” cells from men and women whose genes are more disease-resistant than those of the average person, together with an “instruction manual” to help the body “repair, tweak and improve” its own versions. A single dose could lead to a “body-wide genetic upgrade” that would cut the risk of premature death in some adults by as much as 50 percent.

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I am waiting for tricorders.

The idea of visiting the doctor’s office with symptoms of an illness and leaving with a scientifically confirmed diagnosis is much closer to reality because of new technology developed by researchers at McMaster University.

Engineering, biochemistry and medical researchers from across campus have combined their skills to create a hand-held rapid test for bacterial infections that can produce accurate, reliable results in less than an hour, eliminating the need to send samples to a lab.

Their proof-of-concept research, published today in the journal Nature Chemistry, specifically describes the test’s effectiveness in diagnosing urinary tract infections from real clinical samples. The researchers are adapting the test to detect other forms of bacteria and for the rapid diagnosis of viruses, including COVID-19. They also plan to test its viability for detecting markers of cancer.

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In a study in Nature Plants, Yiping Qi, associate professor of Plant Science at the University of Maryland (UMD), introduces a new and improved CRISPR 3.0 system in plants, focusing on gene activation instead of traditional gene editing. This third generation CRISPR system focuses on multiplexed gene activation, meaning that it can boost the function of multiple genes simultaneously. According to the researchers, this system boasts four to six times the activation capacity of current state-of-the-art CRISPR technology, demonstrating high accuracy and efficiency in up to seven genes at once. While CRISPR is more often known for its gene editing capabilities that can knock out genes that are undesirable, activating genes to gain functionality is essential to creating better plants and crops for the future.

“While my lab has produced systems for simultaneous gene editing [multiplexed editing] before, editing is mostly about generating loss of function to improve the crop,” explains Qi. “But if you think about it, that strategy is finite, because there aren’t endless genes that you can turn off and actually still gain something valuable. Logically, it is a very limited way to engineer and breed better traits, whereas the plant may have already evolved to have different pathways, defense mechanisms, and traits that just need a boost. Through activation, you can really uplift pathways or enhance existing capacity, even achieve a novel function. Instead of shutting things down, you can take advantage of the functionality already there in the genome and enhance what you know is useful.”

In his new paper, Qi and his team validated the CRISPR 3.0 system in rice, tomatoes, and Arabidopsis (the most popular model plant species, commonly known as rockcress). The team showed that it is possible to simultaneously activate many kinds of genes, including faster flowering to speed up the breeding process. But this is just one of the many advantages of multiplexed activation, says Qi.

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A once forgotten technology, RNA editing has been gaining traction as a treatment for genetic conditions given its key advantages over CRISPR gene editing.

Since CRISPR-Cas9 gene editing was first reported in 2012, its promise of making gene editing faster, cheaper, and easier than ever before led to an explosion in the number of publications referring to this gene editing technology.

An increasing number of research labs and companies are aiming to translate CRISPR gene editing into therapies for genetic diseases. However, further research has unveiled that there are more limitations to using CRISPR-Cas9 to cure diseases than initially expected. For example, the technology has been reported to introduce off-target changes to the DNA, raising concerns about its safety.

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Novel study designed to correct genetic abnormalities of red blood cells.

Cleveland Clinic researchers are enrolling patients in a clinical trial that aims to work toward a cure for sickle cell disease, by changing the patient’s genetics. Sickle cell disease, a genetic blood disorder, is a painful and debilitating condition for which there are few approved therapies.

The multicenter study will evaluate the safety and effectiveness of a single dose of EDIT-301, an experimental one-time gene editing cell therapy that modifies a patient’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease.

During the study patients’ stem cells are collected for gene editing in a laboratory. Patients then are treated with chemotherapy to destroy remaining bone marrow to make room for the repaired cells which are infused back into the body. The study will initially enroll 40 adult patients ages 18 to 50 with severe sickle cell disease, with the possibility of expansion to include adolescents. Patients will be monitored closely after treatment for up to two years.

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There are now several monoclonal antibodies, identical copies of a therapeutic antibody produced in large numbers, that are authorized for the treatment of COVID-19. But in the ongoing effort to beat this terrible pandemic, there’s plenty of room for continued improvements in treating infections with SARS-CoV-2, the virus that causes COVID-19.

With this in mind, I’m pleased to share progress in the development of a specially engineered therapeutic antibody that could be delivered through a nasal spray. Preclinical studies also suggest it may work even better than existing antibody treatments to fight COVID-19, especially now that new SARS-CoV-2 “variants of concern” have become increasingly prevalent.

These findings come from Zhiqiang An, The University of Texas Health Science Center at Houston, and Pei-Yong Shi, The University of Texas Medical Branch at Galveston, and their colleagues. The NIH-supported team recognized that the monoclonal antibodies currently in use all require time-consuming, intravenous infusion at high doses, which has limited their use. Furthermore, because they are delivered through the bloodstream, they aren’t able to reach directly the primary sites of viral infection in the nasal passages and lungs. With the emergence of new SARS-CoV-2 variants, there’s also growing evidence that some of those therapeutic antibodies are becoming less effective in targeting the virus.-Dr Francis Collins.

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In America, at least 17 people a day die waiting for an organ transplant. But instead of waiting for a donor to die, what if we could someday grow our own organs?

Last week, six years after NASA announced its Vascular Tissue Challenge, a competition designed to accelerate research that could someday lead to artificial organs, the agency named two winning teams. The challenge required teams to create thick, vascularized human organ tissue that could survive for 30 days.

The two teams, named Winston and WFIRM, both from the Wake Forest Institute for Regenerative Medicine, used different 3D-printing techniques to create lab-grown liver tissue that would satisfy all of NASA’s requirements and maintain their function.

“We did take two different approaches because when you look at tissues and vascularity, you look at the body doing two main things,” says Anthony Atala, team leader for WFIRM and director of the institute.

The two approaches differ in the way vascularization—how blood vessels form inside the body—is achieved. One used tubular structures and the other spongy tissue structures to help deliver cell nutrients and remove waste. According to Atala, the challenge represented a hallmark for bioengineering because the liver, the largest internal organ in the body, is one of the most complex tissues to replicate due to the high number of functions it performs.

Researchers used 3D-printing to create human liver tissue that could soon be tested on the International Space Station.

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