Lifeboat Foundation

The genome editing technology CRISPR has emerged as a powerful new tool that can change the way we treat disease. The challenge when altering the genetics of our cells, however, is how to do it safely, effectively, and specifically targeted to the gene, tissue and organ that needs treatment. Scientists at Tufts University and the Broad Institute of Harvard and MIT have developed unique nanoparticles comprised of lipids—fat molecules—that can package and deliver gene editing machinery specifically to the liver. In a study published today in the Proceedings of the National Academy of Sciences, they have shown that they can use the lipid nanoparticles (LNPs) to efficiently deliver the CRISPR machinery into the liver of mice, resulting in specific genome editing and the reduction of blood cholesterol levels by as much as 57%—a reduction that can last for at least several months with just one shot.

The problem of high cholesterol plagues more than 29 million Americans, according to the Centers for Disease Control and Prevention. The condition is complex and can originate from multiple genes as well as nutritional and lifestyle choices, so it is not easy to treat. The Tufts and Broad researchers, however, have modified one gene that could provide a protective effect against elevated cholesterol if it can be shut down by gene editing.

The gene that the researchers focused on codes for the angiopoietin-like 3 enzyme (Angptl3). That enzyme tamps down the activity of other enzymes—lipases—that help break down cholesterol. If researchers can knock out the Angptl3 gene, they can let the lipases do their work and reduce levels of cholesterol in the blood. It turns out that some lucky people have a natural mutation in their Angptl3 gene, leading to consistently low levels of triglycerides and low-density lipoprotein (LDL) cholesterol, commonly called “bad” cholesterol, in their bloodstream without any known clinical downsides.

Now scientists are hoping to use the knowledge about CRISPR systems to engineering phages to destroy dangerous bacteria.

As the world fights the SARS-CoV-2 virus causing the COVID-19 pandemic, another group of dangerous pathogens looms in the background.

Using an improved version of the gene editing tool CRISPR/Cas9, researchers knocked out up to twelve genes in plants in a single blow. Until now, this had only been possible for single or small groups of genes. The approach was developed by researchers at Martin Luther University Halle-Wittenberg (MLU) and the Leibniz Institute of Plant Biochemistry (IPB). The method makes it easier to investigate the interaction of various genes. The study appeared in The Plant Journal.

The inheritance of traits in plants is rarely as simple and straightforward as Gregor Mendel described. The monk, whose experiments in the 19th century on trait inheritance in peas laid the foundation of genetics, in fact got lucky. “In the traits that Mendel studied, the rule that only one gene determines a specific trait, for example the color of the peas, happened to apply,” says plant geneticist Dr. Johannes Stuttmann from the Institute of Biology at MLU. According to the researcher, things are often much more complicated. Frequently there are different genes that, through their interaction with one another, result in certain traits or they are partly redundant, in other words they result in the same trait. In this case, when only one of these genes is switched off, the effects are not visible in the plants.

The scientists at MLU and IPB have now developed a way to study this complex phenomenon in a more targeted way by improving CRISPR/Cas9. These gene editing tools can be used to cut the DNA of organisms at specific sites. The team built on the work of biologist Dr. Sylvestre Marillonnet who developed an optimized building block for the CRISPR/Cas9 system at the IPB. “This building block helps to produce significantly more Cas9 enzyme in the plants, which acts as a scissor for the genetic material,” explains Stuttmann. The researchers added up to 24 different guide RNAs which guide the scissor enzyme to the desired locations in the genetic material. Experiments on thale cress (Arabidopsis thaliana) and the wild tobacco plant Nicotiana benthamiana proved that the approach works. Up to eight genes could be switched off simultaneously in the tobacco plants while, in the thale cress, up to twelve genes could be switched off in some cases.

In the case of DMD caused by a duplication mutation, CRISPR can simply snip away the harmful duplicate gene, which is much simpler than delivering a new gene or replacing the old.

For the first time in a live animal, researchers have successfully reversed a gene mutation, called a “duplication mutation,” by gene editing.

Summary: A new genetic engineering strategy significantly reduces levels of tau in animal models of Alzheimer’s disease. The treatment, which involves a single injection, appears to have long-last effects.

Source: Mass General.

Researchers have used a genetic engineering strategy to dramatically reduce levels of tau–a key protein that accumulates and becomes tangled in the brain during the development of Alzheimer’s disease–in an animal model of the condition.

The Research Group on Synthetic Biology for Biomedical Applications at Pompeu Fabra University in Barcelona, Spain, has designed a cellular device capable of computing by printing cells on paper. For the first time, they have developed a living device that could be used outside the laboratory without a specialist, and it could be produced on an industrial scale at low cost. The study is published in Nature Communications and was carried out by Sira Mogas-Díez, Eva Gonzalez-Flo and Javier Macía.

We currently have many electronic devices available to us such as computers and tablets whose computing power is highly efficient. But, despite their power, they are very limited devices for detecting biological markers, such as those that indicate the presence of a disease. For this reason, a few years ago ‘biological computers’ began to be developed—in other words, living cellular devices that can detect multiple markers and generate complex responses. In them, the researchers leverage biological receptors that allow detecting exogenous signals and, by means of synthetic biology, modify them to emit a response in accordance with the information they detect.

So far, cellular devices have been developed that must operate in the laboratory, for a limited time, under specific conditions, and must be handled by a specialist in molecular biology. Now, a team of researchers from Pompeu Fabra University has developed new technology to ‘print’ cellular devices on paper that can be used outside the laboratory.

The regeneration of damaged central nervous system (CNS) tissues is one of the biggest goals of regenerative medicine.

Most stroke victims don’t receive treatment fast enough to prevent brain damage. Scientists at The Ohio State University Wexner Medical Center, College of Engineering and College of Medicine have developed technology to “retrain” cells to help repair damaged brain tissue. It’s an advancement that may someday help patients regain speech, cognition and motor function, even when administered days after an ischemic stroke.

Engineering and medical researchers use a process created by Ohio State called tissue nanotransfection (TNT) to introduce genetic material into cells. This allows them to reprogram skin cells to become something different—in this case vascular cells—to help fix damaged brain tissue.

Continue reading “New technology ‘retrains’ cells to repair damaged brain tissue in mice after stroke” »

The development of gene therapy, in particular gene editing using the CRISPR-Cas9 method, has prompted a lively discussion around the world about how deeply you can interfere with the human genome. The creators of this method have turned to the world community, including lawyers, to undertake a public discussion of the implications that it can create (The National Academies of Sciences Engineering Medicine, 2015). The most important problem to be resolved in the future, in my opinion, will be the issue of establishing very clear legal principles of liability for damages resulting from the editing of genes in human embryos and reproductive cells. However, before this happens, it is necessary to show the possible legal problems that may arise and that will certainly appear in future legislative work in the world. Questions must be asked to which world legal experts will need to seek answers. The goal of this paper is to show the possible legal problems and ask questions related to the liability for damages resulting from the editing of genes in human embryos and reproductive cells that will be answered in the future.

Private law considerations will be based on Polish law, although it should be pointed out that the conclusions derived from them appear to be of universal nature for different legal systems. Despite the fact that legal considerations will refer to the regulation of Polish law, the subject of the analysis will also be the differences in the legal qualification of reproductive cells and embryos in other European legislations. It seems that nowhere in the world are there special regulations regarding the liability for damage related to the genetic editing of reproductive cells or embryos. Therefore, there is a need to present new challenges for classic private law institutions, such as legal abilities, torts, or liability for damages. Due to the lack of uniform European regulations and different conflicts of rights the subject of analysis will not be wrongful life and wrongful birth actions, but only claims of prenatal damage to a child.

The first major legal problem facing the international community is, of course, the question of the legal acceptability of the editing of genes of human reproductive cells and embryos (van Dijke et al., 2018). In this regard, it should be pointed out that despite the initial demand to ban such editing, over time, increasingly more scientists have pointed to the fact that it is not possible to maintain such a moratorium (Doudna and Sternberg, 2017). Jiankui’s presentation at the Second International Summit on Human Genome Editing on November 272018, showed that the introduction of a moratorium on genetic modifications of embryos in Europe, the condemnation of such research by a group of 120 of the greatest geneticists, even the Chinese regulations (Zhang and Lie, 2018) will not limit its conduct (Cyranoski and Ledford, 2018). Globalization of the medical market means that if any procedures are allowed on other continents, they will also become available to Europeans (Lunshof, 2016).

In his latest book, Walter Isaacson chronicles the discovery of CRISPR and delves into the ethics of gene editing.