Gene-edited trees are more sustainable and can boost fibre production
17th July 2023
Scientists have used CRISPR gene editing to reduce the lignin content in poplar trees by as much as 50%, offering a potentially more sustainable and efficient method of fibre production.
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Scientists at North Carolina State University (NCSU) have now used a CRISPR gene-editing system to breed poplar trees with reduced levels of lignin, the major barrier to sustainable production of wood fibres, while improving their wood properties. Their study, published in Science, could make fibre production greener, cheaper, and more efficient. This would improve a wide range of consumer items and materials, including paper, cardboard, textiles, hygiene products and more.
Gene therapy eyedrops restored a boy's sight. Similar treatments could help millions
MIAMI (AP) — Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye.
Antonio, who’s been legally blind for much of his 14 years, can see again.
The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. That gave Sabater an idea: What if it could be adapted for Antonio’s eyes?
This insight not only helped Antonio, it also opened the door to similar therapies that could potentially treat millions of people with other eye diseases, including common ones.
Gene therapy targeting sodium successfully reverses chronic pain
By Bronwyn Thompson
July 27, 2023
The National Institute of Health estimates that 100 million Americans, or around a third of the US population, live with a chronic pain condition. While the economic burden, more than US$500 billion annually, is well documented, increasingly studied is its wide-ranging impact on mental health and the growth in opioid addiction.
As such, scientists are eager to develop effective non-opioid, gene-based ways to disrupt the pain signaling experienced in chronic conditions, in the hope that it can vastly improve one's quality of life without any negative side effects.
The latest promising discovery comes out of the New York University (NYU) College of Dentistry’s Pain Research Center, which has developed a novel targeted therapy to alleviate pain delivered by an engineered virus.
Researchers at the University of Bristol have made a remarkable step forward in finding a potential cure for a type of childhood kidney disease.
The research project has shown that just one dose of gene therapy targeting cells in the kidney has the potential to cure a condition known as steroid-resistant nephrotic syndrome.
The findings, which were published in the journal Science Translational Medicine by the team in Bristol led by Professor Moin Saleem, from the Bristol Medical School, suggested that replacing one faulty gene that codes for a protein known as podocin could cure the condition. Podocin is a protein essential for the functioning of cells called podocytes which have a critical role within the kidney's filtration system.
Nephrotic syndrome is a condition where the kidney's filtering units are damaged, allowing large amounts of protein that should be kept in the bloodstream to leak into the urine. This can lead to swelling, particularly in the eyes and legs, and an increased risk of infections and blood clots, and the risk of kidney damage. It can occur at any age but is most commonly diagnosed in children under five years old.
The SMNDC1 gene controls key functions in the human body and is linked to diseases such as diabetes and cancer. Scientists in Stefan Kubicek's research group at the CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences have successfully pinpointed the exact location of the SMNDC1 protein within the cell nucleus and identified an inhibitor that opens up the potential for therapeutic applications of SMNDC1. The study has been published in Nature Communications.
The protein SMNDC1 is considered an essential gene in the human body, present in nearly every cell. Previous studies by Principal Investigator Stefan Kubicek's research group at CeMM had shown that knocking down SMNDC1 can stimulate alpha cells in the islets of Langerhans to produce insulin, potentially offering a new therapeutic target for treating diabetes.
To better understand the function of SMNDC1, the scientists in the Kubicek group investigated its precise cellular location and its interactions with molecules.
SMNDC1 is classified as a splicing factor, meaning it is involved in the process where RNA is transformed into the final messenger RNA that carries the genetic information. This messenger RNA is essentially the blueprint for building a specific protein in a cell. Consequently, SMNDC1 influences the expression of many other proteins.
The clustered regularly interspaced short palindrome repeats (CRISPR) and Crispr-associated protein 9 (CRISPR/Cas9) is now a well-known, revolutionary method to engineer microbial cells.
A key advantage of CRISPR remains in the strain design to facilitate chromosomal integration to enable the assembly of marker-free DNA. These editing systems are highly beneficial; however, their assembly is not quite straightforward and may prevent its use and applications.
In a new report in Nature Communications Biology, Tigran V. Yuzbashev and a research team identified the limits of the existing Cas9 toolkits designed to make CRISPR techniques easier to access and implement. They discussed three different well-established methods and combined them to form a comprehensive toolkit for efficient metabolic engineering by using CRISPR/Cas9.
A single toolkit comprised of 147 plasmids to generate and characterize a library of 137 promoters to build a homogentisic acid in the lab.
Genome modifications with CRISPR/Cas9
The CRISPR/Cas9 system can render quick, precise and scarless genomic modifications to provide significant scope to design microbial strains for bioproduction. Metabolic engineering of yeasts for instance provide a fast-growing area in engineering biology for the sustained production of chemicals, fuels, foods, and pharmaceuticals.
Yeasts have a metabolic potential like eukaryotic cells and are therefore easier to engineer and cultivate at scale. As a result, bioengineers have designed and developed CRISPR systems for yeasts.
CRISPR used to 'reprogram' cancer cells into healthy muscle in the lab
Scientists have transformed cancer cells into healthy muscle tissue in the lab using CRISPR gene-editing technology — and they hope new cancer treatments can be built on the back of this experiment. In a study published Aug. 28 in the journal PNAS, researchers found that disabling a particular protein complex in cells of rhabdomyosarcoma (RMS) — a rare cancer in skeletal muscle tissue that mainly affects children under age 10 — in the laboratory causes the tumor cells to turn into healthy muscle cells.
Although the research is still in its early days, this process of "resetting" cancer cells into healthy cells, broadly known as differentiation therapy, has already been tested in other types of cancer, such as bone and blood cancer. Four drugs have been approved by the U.S. Food and Drug Administration (FDA) to treat the latter disease and generally work by inhibiting a specific protein in the cancer cells.
The protein complex pinpointed in the new research could serve as a target for such a therapy, the study authors wrote, and with further development, it could be a promising new treatment option for patients with RMS, which is normally treated with surgery, radiation and chemotherapy.
"This technology can allow you to take any cancer and go hunting for how to cause it to differentiate," or cause it to stop multiplying uncontrollably and turn into normal, noncancerous cells, Christopher Vakoc, lead author and professor at Cold Spring Harbor Laboratory, said in a statement. "This might be a key step toward making differentiation therapy more accessible."
The gene editing technique CRISPR/Cas9 has allowed researchers to make precise and impactful changes to an organism's DNA to fix mutations that cause genetic disease. However, the CRISPR/Cas9 method can also result in unintended DNA mutations that may have negative effects. Recently, researchers in Japan have developed a new gene editing technique that is as effective as CRISPR/Cas9 while significantly reducing these unintended mutations.
In a new study published in Nature Communications, researchers led by Osaka University introduced a novel technique called NICER, which is based on the creation of multiple small cuts in single DNA strands by an enzyme called a nickase.
New gene-editing tool reduces unintended mutations by more than 70%
By Paul McClure
September 21, 2023
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Researchers have found that splitting the gene editor used in traditional CRISPR technology creates a more precise tool that can be switched on and off, with significantly less chance of causing unintended genome mutations. They say their novel tool can potentially correct around half of the mutations that cause disease.
CRISPR is one of those scientific terms that has made it into the everyday lexicon. Arguably one of the biggest discoveries of the 21st century, the gene-editing tool has revolutionized research and the treatment of genetic and non-genetic diseases. But the primary risk associated with CRISPR technology is ‘off-target edits,’ namely unexpected, unwanted, or even adverse alterations at locations in the genome other than the targeted site.
Now, researchers at Rice University have developed a new CRISPR-based gene-editing tool that’s more precise and significantly reduces the likelihood of off-target edits occurring.
“Our team set out to create a much-improved version that can be turned on or off as needed, providing an unparalleled level of safety and accuracy,” said Hongzhi Zeng, the study’s lead author. “This tool has the potential to correct nearly half of the disease-causing point mutations in our genome. However, current adenine base editors are in a constant ‘on’ state, which could lead to unwanted genome changes alongside the desired correction in the host genome.”
DNA consists of two linked strands that wind around each other, forming a double helix that resembles a twisted ladder. The ‘rungs’ of the ladder are made of base pairs, two complementary nucleotide bases held together by hydrogen bonds: adenine (A) pairs with thymine (T) and cytosine (C) with guanine (G).
