Posts belonging to Category genetics



Gene Researchers Have Created Green Mice

These are no Frankenstein mice. Their green feet come courtesy of a fluorescent green jelly fish gene added to their own genome. This allows a team of British scientists to test out gene editing using CRISPR-Cas9 technology.

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“We take what were or would have been green embryos and we make them into non-green embryos, so it’s a really great way of demonstrating the method“, said Dr. Anthony Perry, reproductive biologist at the University of Bath.

The technique uses the ribonucleic acid molecule CRISPR together with the Cas9 protein enzyme. CRISPR guides the Cas9 protein to a defective part of a genome where it acts like molecular scissors to cut out a specific part of the DNA. This could revolutionise how we treat diseases with a genetic component, like sickle cell anaemia. The technique is being pioneered in the U.S.
We now have a technology that allows correction of a sequence that would lead to normally functioning cells. And I think you know the opportunities with this are really exciting and really profound. There are many diseases that are have known genetic causes that we now have in principle a way to cure,“explains Jennifer Doudna, Professor of cell biology at the University of Berkeley.
Last year two teams of U.S. based scientists used CRISPR-Cas9 technology in mice to correct the genetic mutation that causes sickle cell disease. Although researchers aren’t yet close to using CRISPR-Cas9 to edit human embryos for implantation into the womb – some are already warning against it.

Dr David King, Director of  Human Genetics Alert, comments: “It will immediately create this new form of what we call consumer eugenics, that’s to say eugenics driven by the free market and consumer preferences in which people choose the cosmetic characteristics and the abilities of their children and try to basically enhance their children to perform better than other people’s children.” Other potential applications of the technology could be to make food crops and livestock animal species disease-resistant. The British team say CRISPR-Cas9 presents a golden opportunity to prevent genetic disease.

Source: http://www.reuters.com/
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http://www.bath.ac.uk/

How To Fix Duchenne Muscular Dystrophy

Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes Duchenne muscular dystrophy, a severe muscle-wasting disease. A new study shows that a single injection of CRISPR-Gold, as the new delivery system is called, into mice with Duchenne muscular dystrophy led to an 18-times-higher correction rate and a two-fold increase in a strength and agility test compared to control groups.

Since 2012, when study co-author Jennifer Doudna, a professor of molecular and cell biology and of chemistry at UC Berkeley, and colleague Emmanuelle Charpentier, of the Max Planck Institute for Infection Biology, repurposed the Cas9 protein to create a cheap, precise and easy-to-use gene editor, researchers have hoped that therapies based on CRISPR-Cas9 would one day revolutionize the treatment of genetic diseases. Yet developing treatments for genetic diseases remains a big challenge in medicine. This is because most genetic diseases can be cured only if the disease-causing gene mutation is corrected back to the normal sequence, and this is impossible to do with conventional therapeutics.

CRISPR/Cas9, however, can correct gene mutations by cutting the mutated DNA and triggering homology-directed DNA repair. However, strategies for safely delivering the necessary components (Cas9, guide RNA that directs Cas9 to a specific gene, and donor DNA) into cells need to be developed before the potential of CRISPR-Cas9-based therapeutics can be realized. A common technique to deliver CRISPR-Cas9 into cells employs viruses, but that technique has a number of complications. CRISPR-Gold does not need viruses.

In the new study, research lead by the laboratories of Berkeley bioengineering professors Niren Murthy and Irina Conboy demonstrated that their novel approach, called CRISPR-Gold because gold nanoparticles are a key component, can deliver Cas9 – the protein that binds and cuts DNA – along with guide RNA and donor DNA into the cells of a living organism to fix a gene mutation.

CRISPR-Gold is the first example of a delivery vehicle that can deliver all of the CRISPR components needed to correct gene mutations, without the use of viruses,” Murthy said.

The study was published in the journal Nature Biomedical Engineering.

Source: http://news.berkeley.edu/

Nanogels For Heart Attack Patients

Heart disease and heart-related illnesses are a leading cause of death around the world, but treatment options are limited. Now, one group reports in ACS Nano that encapsulating stem cells in a nanogel could help repair damage to the heart.

Myocardial infarction, also known as a heart attack, causes damage to the muscular walls of the heart. Scientists have tried different methods to repair this damage. For example, one method involves directly implanting stem cells in the heart wall, but the cells often don’t take hold, and sometimes they trigger an immune reaction. Another treatment option being explored is injectable hydrogels, substances that are composed of water and a polymer. Naturally occurring polymers such as keratin and collagen have been used but they are expensive, and their composition can vary between batches. So Ke Cheng, Hu Zhang, Jinying Zhang and colleagues wanted to see whether placing stem cells in inexpensive hydrogels with designed tiny pores that are made in the laboratory would work.

The team encapsulated stem cells in nanogels, which are initially liquid but then turn into a soft gel when at body temperature. The nanogel didn’t adversely affect stem cell growth or function, and the encased stem cells didn’t trigger a rejection response. When these enveloped cells were injected into mouse and pig hearts, the researchers observed increased cell retention and regeneration compared to directly injecting just the stem cells. In addition, the heart walls were strengthened. Finally, the group successfully tested the encapsulated stem cells in mouse and pig models of myocardial infarction.

Source: https://www.acs.org/
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https://global.ncsu.edu/

Rapid, Cheap Liver Cancer Test

University of Utah researchers say they are designing a diagnostic method that will be able to accurately identify signs of liver cancer within minutes, saving critical time for patients of the stealthy disease. The new type of test could forever change how people screen for the disease, said Marc Porter, a U. chemical engineering and chemistry professor who is leading the research along with Dr. Courtney Scaife, a surgeon who both practices and teaches surgery for the university. Porter said the long-term vision is for the tool itself to become as automatic and portable as a pregnancy test, though additional technology — called a spectrometer — is currently needed to precisely measure the results of the test.

A small domino-sized cartridge holds a membrane for a new field test for liver cancer developed by researchers from the University of Utah. The test doesn’t involve sending a specimen to a blood lab and cuts the wait time for results from two weeks to two minutes. It can be administered wherever the patient is, which will be valuable for developing nations with little access to hospitals.

It’s really compact, it’s simple and low cost,” he said of the test kit.

Liver cancer is difficult to survive because typically it is highly developed by the time symptoms show up, Porter said. It is the second deadliest form of cancer worldwide, resulting in about 788,000 deaths in 2015, according to the World Health Organization. “All too often, the cancer is diagnosed past when you can actually have surgical intervention,” Porter said.

Currently, a blood test taken to determine the presence of liver cancer is usually sent to a lab offsite, where it takes days or even up to two weeks to test and return, said Vincent Horiuchi, spokesman for the U.’s College of Engineering. Those days are precious time that is lost in the fight against the disease, he said.

Source: https://unews.utah.edu/

How To Draw Electricity from the Bloodstream

Men build dams and huge turbines to turn the energy of waterfalls and tides into electricity. To produce hydropower on a much smaller scale, Chinese scientists have now developed a lightweight power generator based on carbon nanotube fibers suitable to convert even the energy of flowing blood in blood vessels into electricity.

For thousands of years, people have used the energy of flowing or falling water for their purposes, first to power mechanical engines such as watermills, then to generate electricity by exploiting height differences in the landscape or sea tides. Using naturally flowing water as a sustainable power source has the advantage that there are (almost) no dependencies on weather or daylight. Even flexible, minute power generators that make use of the flow of biological fluids are conceivable. How such a system could work is explained by a research team from Fudan University in Shanghai, China. Huisheng Peng and his co-workers have developed a fiber with a thickness of less than a millimeter that generates electrical power when surrounded by flowing saline solution—in a thin tube or even in a blood vessel.

The construction principle of the fiber is quite simple. An ordered array of carbon nanotubes was continuously wrapped around a polymeric core. Carbon nanotubes are well known to be electroactive and mechanically stable; they can be spun and aligned in sheets. In the as-prepared electroactive threads, the carbon nanotube sheets coated the fiber core with a thickness of less than half a micron. For power generation, the thread or “fiber-shaped fluidic nanogenerator” (FFNG), as the authors call it, was connected to electrodes and immersed into flowing water or simply repeatedly dipped into a saline solution. “The electricity was derived from the relative movement between the FFNG and the solution,” the scientists explained. According to the theory, an electrical double layer is created around the fiber, and then the flowing solution distorts the symmetrical charge distribution, generating an electricity gradient along the long axis.

The power output efficiency of this system was high. Compared with other types of miniature energy-harvesting devices, the FFNG was reported to show a superior power conversion efficiency of more than 20%. Other advantages are elasticity, tunability, lightweight, and one-dimensionality, thus offering prospects of exciting technological applications. The FFNG can be made stretchable just by spinning the sheets around an elastic fiber substrate. If woven into fabrics, wearable electronics become thus a very interesting option for FFNG application. Another exciting application is the harvesting of electrical energy from the bloodstream for medical applications. First tests with frog nerves proved to be successful.

The findings are published in  the journal Angewandte Chemie.

Source: http://newsroom.wiley.com/

China, Global Leader In NanoScience

Mobile phones, computers, cosmetics, bicyclesnanoscience is hiding in so many everyday items, wielding a huge influence on our lives at a microscale level. Scientists and engineers from around the world exchanged new findings and perceptions on nanotechnology at the recent 7th International Conference on Nanoscience and Technology (ChinaNANO 2017) in Beijing last week. China has become a nanotechnology powerhouse, according to a report released at the conference. China’s applied nanoscience research and the industrialization of nanotechnology have been developing steadily, with the number of nano-related patent applications ranking among the top in the world.

According to Bai Chunli, president of the Chinese Academy of Sciences (CAS), China faces new opportunities for nanoscience research and development as it builds the National Center for Nanoscience and Technology  (NCNST) and globally influential national science centers.

We will strengthen the strategic landscape and top-down design for developing nanoscience, which will contribute greatly to the country’s economy and society,” said Bai.

Nanoscience can be defined as the study of the interaction, composition, properties and manufacturing methods of materials at a nanometer scale. At such tiny scales, the physical, chemical and biological properties of materials are different from those at larger scales — often profoundly so.

For example, alloys that are weak or brittle become strong and ductile; compounds that are chemically inert become powerful catalysts. It is estimated that there are more than 1,600 nanotechnology-based consumer products on the market, including lightweight but sturdy tennis rackets, bicycles, suitcases, automobile parts and rechargeable batteries. Nanomaterials are used in hairdryers or straighteners to make them lighter and more durable. The secret of how sunscreens protect skin from sunburn lies in the nanometer-scale titanium dioxide or zinc oxide they contain.

In 2016, the world’s first one-nanometer transistor was created. It was made from carbon nanotubes and molybdenum disulphide, rather than silicon.
Carbon nanotubes or silver nanowires enable touch screens on computers and televisions to be flexible, said Zhu Xing, chief scientist (CNST). Nanotechnology is also having an increasing impact on healthcare, with progress in drug delivery, biomaterials, imaging, diagnostics, active implants and other therapeutic applications. The biggest current concern is the health threats of nanoparticles, which can easily enter body via airways or skin. Construction workers exposed to nanopollutants face increased health risks.

The report was co-produced by Springer Nature, National Center for Nanoscience and Technology (NCNST) and the National Science Library of the Chinese Academy of Sciences (CAS).

Source: http://www.shanghaidaily.com/

Canakinumab Drug Lowers Risk Of Fatal Lung Cancer By 75%

It turns out that cholesterol isn’t the only thing you have to worry about to keep your heart healthy. In recent years, doctors have started to focus on inflammation — the same process that makes cuts red and painful — as an important contributor to a heart attack. It’s the reason doctors recommend low-dose aspirin to prevent recurrent heart attacks in people who have already had them, why they also prescribe statins, which lower both cholesterol and inflammation, and why they have started to measure inflammation levels in the blood.

But it’s never been clear exactly how much inflammation adds to heart disease risk. Since statins lower both, it’s hard to tell whether inflammation or cholesterol has the bigger impact on heart problems. But in a new paper published in the New England Journal of Medicine and presented at the European Society of Cardiology meeting, scientists say they now have proof that lowering inflammation alone, without affecting cholesterol, also reduces the risk of a heart attack.

In the study, 10,000 people who have already had a heart attack were randomly assigned to get injected with a placebo or different doses of a drug called canakinumab. Canakinumab, made by Novartis, is currently approved to treat rare immune-related conditions and works to reduce inflammation but does not affect cholesterol levels. After four years, the people who received the drug had a 15% lower chance of having a heart attack or stroke compared to people who didn’t get the drug. The medication also reduced the need for angioplasty or bypass surgery by 30%.

Even I am pinching myself,” says Dr. Paul Ridker, who led the study and is director of the center for cardiovascular disease prevention at Brigham and Women’s Hospital and is a pioneer in exposing the role inflammation plays in heart disease. “This outcome is more than we hoped for. The bottom line is we now have clear evidence that lowering inflammation through this pathway lowers rates of heart attack and stroke with no change at all in cholesterol.”

Perhaps more intriguing are additional results that Ridker reported, related to cancer. In a separate study published in the Lancet using data from the same study, he found that people taking canakinumab lowered their risk of dying from any cancer over four years by 50%, and their risk of fatal lung cancer by 75%.

Source: http://time.com/

Brain Cells Found To Control Aging

Scientists at Albert Einstein College of Medicine have found that stem cells in the brain’s hypothalamus govern how fast aging occurs in the body. The finding, made in mice, could lead to new strategies for warding off age-related diseases and extending lifespan. The hypothalamus was known to regulate important processes including growth, development, reproduction and metabolism. In a 2013 Nature paper, Einstein researchers made the surprising finding that the hypothalamus also regulates aging throughout the body. Now, the scientists have pinpointed the cells in the hypothalamus that control aging: a tiny population of adult neural stem cells, which were known to be responsible for forming new brain neurons.

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Our research shows that the number of hypothalamic neural stem cells naturally declines over the life of the animal, and this decline accelerates aging,” says senior author Dongsheng Cai, M.D., Ph.D., professor of molecular pharmacology at Einstein. “But we also found that the effects of this loss are not irreversible. By replenishing these stem cells or the molecules they produce, it’s possible to slow and even reverse various aspects of aging throughout the body.”

The findings have been published online in Nature.

Source: http://www.einstein.yu.edu/
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http://www.reuters.com/

Male Unfertility Rises Sharply In Developed World

Male fertility in the developed world is in sharp decline. A new study from the Hebrew University of Jerusalem shows a 52.4 percent fall in sperm concentration While total sperm count fell 59.3 percent between 1973 and 2011.

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Our findings of sharp decline in sperm count among western men is the canary in the coal mine. It signifies that we have a serious problem with the health of men in the western world,” says Hagai Levine, lead-researcher at Hebrew University-Hadassah School of Public Health.

That’s because sperm count is a marker of men’s general health as well as fertility. The study analysed sperm count studies from across the world – and the trend was reflected in America, Europe, Australia and New Zealand. The next step is to investigate the causes of male infertility.
From previous research we know that exposure to man-made chemicals, especially during the critical period of the development of the male reproductive system in pre-natal life, in the early stages of pregnancy can severaly disrupt and can manifest later in life as low sperm count and problems with male fertility,” explains Hagai Levine. The study controlled for factors like age, sexual activity and the types of men, making its conclusions more reliable. “So if, for example, you have 50 studies in one country and they all show the same trend in declining sperm counts, including different counting methods in different groups of men, that makes it much more likely that it’s real” states Prof. Daniel Brison, scientific Director at the University of Manchester (Dept. of Reproductive Health).

The decline shows no sign of slowing. And the researchers say further research is urgently needed – and regulation of the environmental factors that may be contributing could be part of the solution.

Source: https://academic.oup.com/
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http://www.reuters.com/

Faulty DNA Linked To Fatal Heart Condition Removed From Embryo

Scientists have modified human embryos to remove genetic mutations that cause heart failure in otherwise healthy young people in a landmark demonstration of the controversial procedure. It is the first time that human embryos have had their genomes edited outside China, where researchers have performed a handful of small studies to see whether the approach could prevent inherited diseases from being passed on from one generation to the next.

While none of the research so far has created babies from modified embryos, a move that would be illegal in many countries, the work represents a milestone in scientists’ efforts to master the technique and brings the prospect of human clinical trials one step closer. The work focused on an inherited form of heart disease, but scientists believe the same approach could work for other conditions caused by single gene mutations, such as cystic fibrosis and certain kinds of breast cancer.

This embryo gene correction method, if proven safe, can potentially be used to prevent transmission of genetic disease to future generations,” said Paula Amato, a fertility specialist involved in the US-Korean study at Oregon Health and Science University.

The scientists used a powerful gene editing tool called Crispr-Cas9 to fix mutations in embryos made with the sperm of a man who inherited a heart condition known as hypertrophic cardiomyopathy, or HCM. The disease, which leads to a thickening of the heart’s muscular wall, affects one in 500 people and is a common cause of sudden cardiac arrest in young people. Humans have two copies of every gene, but some diseases are caused by a mutation in only one of the copies. For the study, the scientists recruited a man who carried a single mutant copy of a gene called MYBPC3 which causes HCM.

Source: https://www.theguardian.com/

New Brain Death Pathway In Alzheimer’s Identified

Findings of team led by the Arizona State University (ASU) scientists offer hope for therapies targeting cell loss in the brain, an inevitable and devastating outcome of Alzheimer’s progression
Alzheimer’s disease tragically ravages the brains, memories and, ultimately, personalities of its victims. Now affecting 5 million Americans, Alzheimer’s disease is the sixth-leading cause of death in the U.S., and a cure for Alzheimer’s remains elusive, as the exact biological events that trigger it are still unknown.

In a new study, Arizona State University-Banner Health neuroscientist Salvatore Oddo and his colleagues from Phoenix’s Translational Genomics Research Institute (TGen) — as well as the University of California, Irvine, and Mount Sinai in New York — have identified a new way for brain cells to become fated to die during Alzheimer’s disease. The research team has found the first evidence that the activation of a biological pathway called necroptosis, which causes neuronal loss, is closely linked with Alzheimer’s severity, cognitive decline and extreme loss of tissue and brain weight that are all advanced hallmarks of the disease.

We anticipate that our findings will spur a new area of Alzheimer’s disease research focused on further detailing the role of necroptosis and developing new therapeutic strategies aimed at blocking it,” said Oddo, the lead author of this study, and scientist at the ASU-Banner Neurodegenerative Disease Research Center at the Biodesign Institute and associate professor in the School of Life Sciences.

Necroptosis, which causes cells to burst from the inside out and die, is triggered by a triad of proteins. It has been shown to play a central role in multiple sclerosis and Lou Gehrig’s disease (amyotrophic lateral sclerosis, or ALS), and now for the first time, also in Alzheimer’s disease.

There is no doubt that the brains of people with Alzheimer’s disease have fewer neurons,” explained Oddo. “The brain is much smaller and weighs less; it shrinks because neurons are dying. That has been known for 100 years, but until now, the mechanism wasn’t understood.
The findings appear in the advanced online edition of Nature Neuroscience.

Source: https://asunow.asu.edu/

Cancer: A Giant Step For Immunotherapy

A Food and Drug Administration (FDA) panel opened a new era in medicine, unanimously recommending that the agency approve the first-ever treatment that genetically alters a patient’s own cells to fight cancer, transforming them into what scientists call “a living drug” that powerfully bolsters the immune system to shut down the disease.

If the F.D.A. accepts the recommendation, which is likely, the treatment will be the first gene therapy ever to reach the market. Others are expected: Researchers and drug companies have been engaged in intense competition for decades to reach this milestone. Novartis is now poised to be the first. Its treatment is for a type of leukemia, and it is working on similar types of treatments in hundreds of patients for another form of the disease, as well as multiple myeloma and an aggressive brain tumor.

To use the technique, a separate treatment must be created for each patient — their cells removed at an approved medical center, frozen, shipped to a Novartis plant for thawing and processing, frozen again and shipped back to the treatment center.

A single dose of the resulting product has brought long remissions, and possibly cures, to scores of patients in studies who were facing death because every other treatment had failed. The panel recommended approving the treatment for B-cell acute lymphoblastic leukemia that has resisted treatment, or relapsed, in children and young adults aged 3 to 25.

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We believe that when this treatment is approved it will save thousands of children’s lives around the world,” Emily’s father, Tom Whitehead, told the panel. “I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive.”

The main evidence that Novartis presented to the F.D.A. came from a study of 63 patients who received the treatment from April 2015 to August 2016. Fifty-two of them, or 82.5 percent, went into remission — a high rate for such a severe disease. Eleven others died.

It’s a new world, an exciting therapy,” said Dr. Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society, which paid for some of the research that led to the treatment. The next step, she said, will be to determine “what we can combine it with and is there a way to use it in the future to treat patients with less disease, so that the immune system is in better shape and really able to fight.” She added, “This is the beginning of something big.”

Source: http://www.chop.edu/
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