CRISPR & Genetic Engineering News and Discussions

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Researchers develop messenger RNA therapy for ovarian cancer, muscle wasting
https://phys.org/news/2022-09-messenger ... ancer.html
by Steve Lundeberg, Oregon State University

Researchers at Oregon State University and Oregon Health & Science University have developed a promising, first-of-its-kind messenger RNA therapy for ovarian cancer as well as cachexia, a muscle-wasting condition associated with cancer and other chronic illnesses.

The treatment is based on the same principles used in SARS-CoV-2 vaccines, and the scientists say mRNA technology, though still in its infancy in terms of therapeutic application, holds tremendous clinical potential for the management of disease. Messenger RNA carries instructions to cells regarding the manufacture of proteins.

The findings, achieved through a mouse model and published today in the journal Small, are important because ovarian cancer is a particularly deadly form of cancer, with a five-year survival rate of less than 30% if it has spread beyond the ovaries.
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Gene-edited tomato can fight cancer and heart disease

13th September 2022

U.S. regulators have approved a new purple tomato, genetically engineered to be packed with antioxidants and anthocyanins. The fruit will go on sale in 2023.

[...]

U.S. home growers should be able to purchase seeds and grow the enhanced tomato from spring 2023.

[...]

A study published in the peer-reviewed journal Nature Biotechnology showed that mice fed a diet supplemented with the high-anthocyanin tomatoes achieved a significant extension of lifespan.

https://www.futuretimeline.net/blog/202 ... tomato.htm


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Xyls
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Scientists hail autoimmune disease therapy breakthrough

https://www.theguardian.com/science/202 ... cell-lupus
Five people with severe autoimmune disease have become the first in the world to receive a groundbreaking therapy that uses genetically altered cells to drive the illness into remission.

The four women and one man, aged 18 to 24, received transfusions of modified immune cells to treat severe lupus, an autoimmune disease that can cause life-threatening damage to the heart, lungs, brain and kidneys.
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Researchers discover dozens of genetic defects important for immune defense and relevant for patients with rare diseases
https://medicalxpress.com/news/2022-09- ... mmune.html
by University of Helsinki

Researchers from the Institute of Biotechnology, University of Helsinki, pioneers in identifying the first patient mutations on the NFkB1-gene, cooperated with international clinicians to identify and characterize a plethora of unreported NFKB1 variants on patients with immune system related illnesses.

In many cases, the identification of a genetic defect in a patient is of great importance for the treatment and prognosis of patients with rare diseases. NFKB1, a transcription factor, causes changes in gene expression and is activated by stress and immune related signaling pathways. Mutations in the NFkB1 have previously been linked to common variable immune deficiency (CVID).

Two new studies published in Frontiers in Immunology may bring further relief for patients with hereditary gene defects in their immune systems.

"These studies have significantly expanded the associations of NFKB1 variants to immune system dysfunction—the connections which we first reportedin 2017," says research director Markku Varjosalo from the Institute of Biotechnology, University of Helsinki

Researchers identified two new NFKB1 variants in two families suffering from common variable immune deficiency. Both identified NFKB1 variants caused reduced expression of the NFKB1 protein and lead to an altered gene expression and increased inflammation response in patient cells. Interaction analysis again showed loss of interactions for one of the variants but not the other.
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Scientists engineer mosquitoes that can't spread malaria
https://phys.org/news/2022-09-scientist ... laria.html
by Imperial College London
Scientists have engineered mosquitoes that slow the growth of malaria-causing parasites in their gut, preventing transmission of the disease to humans.

The genetic modification causes mosquitoes to produce compounds in their guts that stunt the growth of parasites, meaning they are unlikely to reach the mosquitoes' salivary glands and be passed on in a bite before the insects die.

So far, the technique has been shown to dramatically reduce the possibility of malaria spread in a lab setting, but if proven safe and effective in real-world settings it could offer a powerful new tool to help eliminate malaria.

The innovation, by researchers from the Transmission:Zero team at Imperial College London, is designed so it can be coupled with existing "gene drive" technology to spread the modification and drastically cut malaria transmission. The team is looking towards field trials, but will thoroughly test the safety of the new modification before combining it with a gene drive for real-world tests.

Collaborators from the Institute for Disease Modeling at the Bill and Melinda Gates Foundation also developed a model that, for the first time, can assess the impact of such modifications if used in a variety of African settings. They found that the modification developed by the Transmission:Zero team could be a powerful tool for bringing down cases of malaria even where transmission is high.

The results of the modification technology in the lab and the modeling are published today in Science Advances.
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New RNA-based tool can illuminate brain circuits, edit specific cells
https://phys.org/news/2022-10-rna-based ... cuits.html
by Duke University

Duke University researchers have developed an RNA-based editing tool that targets individual cells, rather than genes. It is capable of precisely targeting any type of cell and selectively adding any protein of interest.

Researchers said the tool could enable modifying very specific cells and cell functions to manage disease.

Using an RNA-based probe, a team led by neurobiologist Z. Josh Huang, Ph.D. and postdoctoral researcher Yongjun Qian, Ph.D. demonstrated they can introduce into cells fluorescent tags to label specific types of brain tissue; a light-sensitive on/off switch to silence or activate neurons of their choosing; and even a self-destruct enzyme to precisely expunge some cells but not others. The work appears Oct. 5 in Nature.

Their selective cell monitoring and control system relies on the ADAR enzyme, which is found in every animal's cells. While these are early days for CellREADR (Cell access through RNA sensing by Endogenous ADAR), the possible applications appear to be endless, Huang said, as is its potential to work across the animal kingdom.

"We're excited because this provides a simplified, scalable and generalizable technology to monitor and manipulate all cell types in any animal," Huang said. "We could actually modify specific types of cell function to manage diseases, regardless of their initial genetic predisposition," he said. "That's not possible with current therapies or medicine."
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New gene-editing platform broadens scope of use of CRISPR gene editing
https://phys.org/news/2022-10-gene-edit ... rispr.html
by Win Reynolds, Northwestern University
A team of researchers at Northwestern University has devised a new platform for gene editing that could inform the future application of a near-limitless library of CRISPR-based therapeutics.

Using chemical design and synthesis, the team brought together the Nobel-prize winning technology with therapeutic technology born in their own lab to overcome a critical limitation of CRISPR. Specifically, the groundbreaking work provides a system to deliver the cargo required for generating the gene editing machine known as CRISPR-Cas9. The team developed a way to transform the Cas-9 protein into a spherical nucleic acid (SNA) and load it with critical components as required to access a broad range of tissue and cell types, as well as the intracellular compartments required for gene editing.

The research, published today in a paper titled "CRISPR Spherical Nucleic Acids," in the publication Journal of the American Chemical Society, and shows how CRISPR SNAs can be delivered across the cell membrane and into the nucleus while also retaining bioactivity and gene editing capabilities.

The work builds on a 25-year effort steered by nanotechnology pioneer Chad A. Mirkin, who led the study, to uncover the properties of SNAs and the factors that distinguish them from their well-known linear cousin, the blueprint of life. He is famed for his invention of SNAs, structures typically comprised of spherical nanoparticles densely covered with DNA or RNA, giving them chemical and physical properties radically different from those forms of nucleic acids found in nature.
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A new technique for observing gene activity in a test tube
https://phys.org/news/2022-10-technique-gene-tube.html
by Julius-Maximilians-Universität Würzburg
When searching for the causes of illnesses and developing new treatments, it is absolutely vital to have a precise understanding of the genetic fundamentals. Würzburg researchers have devised a new technique for this purpose.

Pathological processes are usually characterized by altered gene activity in the cells affected. So, gaining an accurate picture of gene activity can provide the key to the development of new, targeted therapies. Whether these therapies then work as we would want them to can also be verified by looking at genes and the processes they initiate.

It is no wonder that research is focused on methods and techniques that provide detailed information about the genetic activity of individual cells. A research team at the University of Würzburg (JMU) has now developed a technique that is a significant improvement on the methods used to date. Scientists from the Institute for Molecular Infection Biology (IMIB) and the Helmholtz Institute for RNA-based Infection Research (HIRI) were involved. They have presented the results of their work in the current issue of the journal Nucleic Acids Research.

Analysis of a synthetic transcriptome

"We have developed a technique that can be used to analyze the translational landscape of a fully customizable synthetic transcriptome, in other words one outside the cell," is how Jörg Vogel explains the central outcome of the study. Vogel heads the Institute for Molecular Infection Biology at JMU and is also the Director of HIRI as well as the principal author of the study. The new technique has been given the scientific name INRI-seq, which is short for in vitro Ribo-seq.
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Gene correction as a possible therapy for iron storage disease
https://medicalxpress.com/news/2022-10- ... sease.html
by Stefan Zorn, Hannover Medical School
Hereditary primary haemochromatosis is one of the most common inborn errors of metabolism in Europe. In this disorder, also known as iron storage disease, the body is overloaded with iron. The excess iron accumulates in organs and tissues and leads to slowly progressive damage to the liver, heart, pancreas, pituitary gland and joints. This can lead to changes in the heart muscle (cardiomyopathies) or diabetes mellitus (bronchial diabetes), and even to scarring of the liver tissue (liver cirrhosis) and liver cancer.

The cause is a genetic defect that disrupts the regulation of iron absorption via the mucous membrane of the small intestine. A research team led by Professor Dr. Michael Ott and Dr. Simon Krooss from the Department of Gastroenterology, Hepatology and Endocrinology at the Hannover Medical School (MHH) has now found a way to treat the hereditary disease with the help of targeted gene correction. The work has been published in the journal Nature Communications.

Control of iron absorption defective

"In most cases, iron storage disease is due to a defect in the haemochromatosis gene HFE, which is located on chromosome 6," says Professor Ott. It only occurs in people who have inherited this defect from both parents, i.e. who do not have a "healthy" gene to compensate. In more than 80% of those affected, a certain change, called the C282Y mutation, is found in both copies of the HFE gene. This leads to the replacement of an amino acid—i.e. a protein building block—in the HFE protein.
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Proof-of-concept study advances potential new way to deliver gene therapy
https://phys.org/news/2022-10-proof-of- ... erapy.html
by Johns Hopkins University School of Medicine
Johns Hopkins Medicine researchers say they have successfully used a cell's natural process for making proteins to "slide" genetic instructions into a cell and produce critical proteins missing from those cells. If further studies verify their proof-of-concept results, the scientists may have a new method for targeting specific cell types for a variety of disorders that could be treated with gene therapies. Such disorders include neurodegenerative diseases that affect the brain, including Alzheimer's disease, forms of blindness and some cancers.

For those looking to develop treatments for diseases where cells lack a specific protein, it's critical to precisely target the cell causing the disease in each structure, such as the brain, to safely kickstart the protein-making process of certain genes, says Seth Blackshaw, Ph.D., professor of neuroscience in the Sol Snyder Department of Neuroscience and member of the Institute for Cell Engineering at the Johns Hopkins University School of Medicine. Therapies that don't precisely target diseased cells can have unintended effects in other healthy cells, he adds.

Two methods currently used to deliver protein-making packages into cells vary widely in their effectiveness in both animal models and people. "We wanted to develop a gene expression delivery tool that's broadly useful in both preclinical and clinical models," says Blackshaw.

One current method of sending biochemical packages involves so-called "mini promoters" that direct the expression (protein-making process) of certain stretches of DNA. Blackshaw says this method often fails to express genes in the right cell type.
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