Those results raised concerns about the use of CRISPR technology in studying human diseases. As a potential solution
Now, a team of researchers from Duke university have shown that these gene-controlling methods are capable of the high degree of precision required for basic science and medical research.
and potentially treating human diseases such as cancer, cardiovascular disease, neurodegenerative conditions and diabetes, which can be driven by mutations in control regions of the genome.
The hope is that overriding one of these switches could uncover and fix the root causes of many diseases.
It could also help researchers understand and change how different people respond to drugs. But only if the CRISPR technique is specific enough.
how those switches differ between individuals and the implications of these insights for human traits and diseases.
Crawford, associate professor of pediatrics, has spent more than a decade developing techniques to identify control regions across the genome
The discovery was published electronically in the journal Ophthalmology. his is the culmination of more than 20 years of work,
first mapped the gene causing North carolina macular dystrophy on chromosome 6 in 1992. The current findings ultimately required an international team of 20 investigators using data from the Human genome Project
and an elaborate computer analysis to identify the actual mutations in INTERGENIC DNA near the PDRM13 gene. ndividuals with this disease have normal eyes except that they fail to form maculas,
Small says. nderstanding how this gene works may help us treat many macular diseases more effectively in the future. cientists already know how to create new retinal cells from a patient skin,
Ltd. s a person who knows firsthand what it is like to lose vision from a rare, inherited eye disease,
and I feel that the prospect of finding a cure is possible and probable in the short term
and certain in the long term. s a public research university working to solve some of society greatest health and medical challenges,
says Jean Robillard, M d.,interim president of the University of Iowa and vice president for medical affairs, University of Iowa Health care s
which is the leading cause of serious long-term disability in adults. The five-year study, performed in an animal model,
and programs in the UCLA department of neurology. he brain has limited a capacity for recovery after stroke,
and to RNA in the brain cells of people with other diseases. They found that GDF10 regulates a unique collection of molecules that improves recovery after stroke.
and Health Conference, being held this week at the University of North carolina School of Global Public health.
The technology transfer company will produce ceramic water disinfection tablets called adidropsfor people in developing countries who have poor access to clean drinking water.
killing pathogens by penetrating cell membranes and disrupting cell division. Extensive testing at UVA labs show that the tablet causes better than a 99.99 percent reduction in such infectious waterborne bacteria as Vibrio cholera
Escherichia coli and other coliform bacteria. The Madidrop also is effective at reducing the infectivity of protozoan pathogens including Giardia lamblia and Cryptosporidium parvum, all of
which cause severe diarrhea, vomiting, dehydration and potential long-term growth and cognition deficiencies. These pathogens are particularly devastating to children and people with AIDS.
Smith said that most of the revenue from sales of Madidrop initially will be fed back into the company to fuel production expansion.
Public service-minded investors will become shareholders and eventually make small profits. Madidrop PBC will provide profit-sharing for employees
Smith said. e are actively raising additional capital to help us bring this innovative public health product to people in need around the world. adidrop PBC administrative office is located on Allied Street in Charlottesville
said Robert Mckenna, Ph d.,a professor of biochemistry and molecular biology in the UF College of Medicine,
San diego, have developed a method that cuts down by half the time needed to make high-tech flexible sensors for medical applications.
Coleman team at UC San diego has been working in medical settings for four years. Their sensors have been used to monitor premature babies, pregnant women,
patients in Intensive care units and patients suffering from sleep disorders. Coleman and colleagues quickly found out that nurses wanted the sensors to come in a peel-and-stick form,
like a medical-grade Band aid. The medium on which the sensors were placed also needed to be approved FDA.
the researchers published their findings in the Proceedings of the National Academy of Sciences. hen you have an infection,
it can be very difficult for a doctor to know which bacteria is infecting you,
A doctor may try one class, and if that doesn work, try another class. We need more broad-spectrum antimicrobial agents.
Such structures have been investigated for various medical applications but because they do not like water, they do not travel well in bodily fluids.
#Nanotechnology could spur new heart treatment for arrthymia A new nanoparticle developed by University of Michigan researchers could be the key to a targeted therapy for cardiac arrhythmia,
and can lead to heart attack and stroke. The disease affects more than four million Americans and causes over 750,000 hospitalizations and 130,000 deaths per year in the United states alone.
The new treatment uses nanotechnology to precisely target and destroy the cells within the heart that cause cardiac arrhythmia.
Their findings are detailed in a new paper published in the journal Science Translational Medicine.
Today, the disease is treated usually with drugs, which can have serious side effects. It can also be treated with a procedure called cardiac ablation that burns away the malfunctioning cells using a high-powered laser that threaded into the heart on a catheter.
The team, led by Jérôme Kalifa, M d.,Ph d.,a cardiologist and U-M Medical school assistant professor at the Center for Arrhythmia Research,
Widely used today to treat cancer, the technique requires doctors to mark unwanted cells with a chemical that makes them sensitive to low-level red light.
The red light then destroys the marked cells while leaving surrounding tissue unharmed. he great thing about this treatment is that it precise down to the level of individual cells,
The major challenge of adapting the therapy to heart cells was developing a nanoparticle small enough to penetrate the tiny pores inside heart capillaries,
yet large enough to carry the chemical payload needed to do the job. n our cancer work,
and a research lab specialist in internal medicine. his cell-selective therapy may represent an innovative concept to overcome some of the current limitations of cardiac ablation,
#Cell stress response and fat and obesity gene linked In one fell swoop, Cornell researchers have discovered mechanisms that control the function of a fat
and obesity gene while at the same time answering a longstanding question about how cells respond to stress.
when they have fevers. The genes express heat shock proteins which protect essential cell proteins and remove damaged proteins before they accumulate
disease and cell death. eat shock genes and their heat shock proteins are expressed highly during stress and are very critical to protect cells;
without them cells would quickly die under stress, said Shu-Bing Qian, associate professor of nutritional sciences,
along with Dr. Samie Jaffrey, professor of pharmacology at Weill Cornell Medicine. Jun Zhou, a postdoctoral research associate in Qian lab, is the paper first author.
and it is associated also with diabetes, he said. The gene produces an FTO enzyme, which acts as a demethylase,
Many years ago, researchers observed that obese people have poor cellular stress responses. This may be because their heat shock proteins are reduced,
whether FTO enzymes and their effect on heat shock genes and proteins could be promoting obesityand diabetes,
Drugs that flip this switch rapidly reduced obesity and diabetes risk factors in mice fed a high fat diet.
The results suggest that drugs capable of targeting similar molecular pathways in human fat cells could one day become major tools for fighting the growing worldwide epidemics of obesity and type 2 diabetes, according to senior
He holds a joint appointment in UCSF Diabetes Center and Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research.
white, which stores energy and is linked with diabetes and obesity; and brown, which produces heat by burning energy
which is already in clinical trials as a cancer therapeutic; and a more precise next-generation antisense oligonucleotide (ASO) drug developed in collaboration with Isis pharmaceuticals,
but here wee found that it could help reduce the risk of type 2 diabetes, as well.
genetics and disease diagnosis. But carrying out such analyses requires expensive lab equipment, making its application out of reach for many people who live in resource-limited places.
After only a 10-minute run, the device could detect the Hepatitis b virus in blood serum at a level low enough to flag an early-stage acute infection,
Another real-world use for magnetic field cloaking would be medicine. Magnetic resonance imaging or MRI, utilizes magnetic fields. A cloaking device for magnetic fields could make it easier for doctors to utilize MRI during operations by idingthe field from surgical instruments. hat just one application.
But who knows what other applications there could be for this theory, which works for almost any kind of wave, said Uhlmann.
a superconducting sphere and a magnetic sheet that had been wound into a cylinder. The metamaterial had just the right properties to cloak a specific magnetic field encased within the sphere,
and eggs has lead to numerous breakthroughs in reproductive health, and now he believes his team has discovered a revolutionary strategy to treat cancer. he focus of my work has always been to define the signature molecules that are on
and within the egg and the sperm, said Herr, a professor of cell biology in the School of medicine.
and his team create using monoclonal antibodies antibodies designed to bind to proteins originating in only one type of cell.
or to the sperm gives you opportunities to create small-molecule drugs for female and male contraception, contraceptive vaccines,
This vastatinbirth control method would be free of unpleasant side-effects like mood swings, acne and breast tenderness,
and previously unknown, characteristic of cancerous tumors arising from a variety of organs. e discovered membrane proteins that among the normal tissues,
These egg-specific proteins are also found in cancers that arise in a wide range of organs,
For some reason, when many cancers dysregulate, or begin to grow on their own, they revert back and take on features of a developing egg.
Herr and his team are bringing awareness to an aspect of cancer that is fundamental to the disease:
At the same time, theye creating an entirely new field of study into cancer-oocyte neoantigens. athologists have appreciated long that cancer cells may de-differentiate
What we have confirmed now is that cancers from many organs de-differentiate to take on features of the oocyte, the original mother cell from
With this discovery, Herr started his first cancer research company, Neoantigenics. Like Ovastasis, Neoantigenics is focused on creating a targeted drug that will affect only those cells identified by the correct cell surface biomarkers.
Their goal is to create a cancer treatment that will track and kill cells that carry the SAS1B protein,
the biomarker that is found only on growing eggs and tumor cells. The monoclonal antibodies to SAS1B can be thought of as a homing mechanism to guide a miniscule warhead selectively to the surface of cancer cells.
and then after binding on the tumor cell surface, the antibody-drug burrows inside them to release a toxic payload. ou add an antibody with a drug on it
and within 15 minutes of contacting the cancer cells, it binds at the cell surface and begins the internalization process,
After about an hour, the SAS1B-marked antibodies reach compartments inside the cell and release the drug payload, triggering changes that result in cell death within a few days.
The same biomarkers that will help limit the area of impact for Ovastasis birth control will also help Neoantigenics confine the toxic effects of cancer treatment to growing egg and tumor cells.
This unique medication could mean a dramatic reduction of the difficult side-effects of traditional cancer treatments like hair loss, nausea, anemia and neuropathy.
but for female cancer patients especially, a treatment that doesn touch their body healthy tissues is a huge breakthrough. e think we have a way
but a way that could become a frontline treatment for women who have cancers of many types
After the cancer treatment is complete those primordial eggs can begin the cycle of normal egg growth and ovulation again.
and the company will work with U. Va. labs as it begins testing the cancer medication first in model organisms,
so may find applications in drug toxicity tests, the search for new drugs and cell therapy.
making them useful for applications in drug discovery and toxicology. This study was supported by CREST program of Japan Agency for Medical Research and development,
and Grants-in-Aid for Scientific research of Japan Society for the Promotion of Science f
The course of infection, from exposure to recovery, or death, can take as little as two weeks. That may not leave enough time for the immune system to mount an effective response.
The goal of some antiviral therapies, therefore, is to buy more and give the immune system a leg up on the virus. A new study led by Bruce Freedman
but also Marburg, Lassa and Junin viruses, all sources of deadly infections. The work paves the way toward designing a potential broad-spectrum drug that could serve as a therapy for a number of serious viral infections. ur work is aimed at handicapping the virus
so that the immune system has time to respond, said Freedman. e could also imagine this type of drug would be part of a cocktail therapy,
like those used for HIV, to cripple the virus at different stages of its life cycle,
The research was published in the journal PLOS Pathogens. Freedman and Harty teamed with scientists from Fox Chase Chemical Diversity Center and the United states army Medical Research Institute of Infectious disease on the study.
Because viruses must hijack host cell proteins to complete their life cycle and reproduce, the Penn Vet scientists have focused on developing drugs that interfere with the host proteins
and found that it lowered infection rates in a dose-dependent manner. Finally, they confirmed that ORAI1 inhibition was blocking the ability of viruses to exit the cell,
Freedman and Harty have launched a company called Intervir Therapeutics Inc.,with support from the Penn Center for Innovation UPSTART program,
and minimize any toxic effects. They noted that such drugs would only be given for a short duration,
In addition, if used as part of a combination therapy, ORAI inhibitors could be administered at lower doses m
Future applications of such a DNA walker might include a cancer detector that could roam the human body searching for cancerous cells
constantly computing whether a cancer is present. ore immediate practical applications may include deploying the DNA walker in the body
and targeted by doctors. There also may be implications for future delivery of nanoscale therapeutics. Although it may be a long march from diagnosing cancer to curing it,
ll breakthroughs begin with baby steps. Only in this case they are the steps of a DNA walker,
said co-author Jung. The walker is made from a single piece of DNA with two legs connected by a torso.
and smuggle cancer signals their neighbors New research in The FASEB Journal suggests that an in vitro co-culture system robustly quantifies the transfer of fluorescent proteins between cells
Not only does this cast an important light on how cancer metastasizes and recruits cellular material from healthy cells,
but it also suggests that these physical channels might be exploitable to deliver drug therapies. hope that the tools we have developed,
especially the mouse model, will be used by academics to isolate healthy cells modified by tumors, and by the pharmaceutical industry in the quest for novel anticancer drugs that block tumor-organ communication,
said Anne Burtey, Ph d.,study author from the Department of Biomedicine, at the University of Bergen in Bergen,
Norway. also hope the knowledge we provide here is paving the way to engineer uper-spreadingagents,
with increased abilities to diffuse within tumors and even reach the healthy cells involved in tumor progression.
To make this discovery, Burtey and colleagues studied the exchange of molecules between cells, by color-coding them with red or blue cellular fluorescent yesor ags.
suggesting that this protein is a key regulator of cell-cell communication in cancer. Live cell imaging confirmed that the transfer is contact-dependent.
or use them to deliver lifesaving therapies. o
#New treatment targets cancers with particular genetic signature Oxford university researchers have found the Achilles heel of certain cancer cells mutations in a gene called SETD2.
Their findings will be presented to the National Cancer Research Institute conference in Liverpool 2nd november, 2015. It is well known that mutations drive cancer cell growth and resistance to treatment.
However, these mutations can also become a weak point for a tumour. The Oxford team found that that was the case for cancer cells with mutations in a key cancer gene called SETD2.
Study author, Dr Timothy Humphrey said: utations in SETD2 are frequently found in kidney cancer and some childhood brain tumours,
so we were excited when we discovered that a new drug we were studying specifically killed cancer cells with this mutation.
WEE1 was discovered first by British Nobel prize winner Sir Paul Nurse. The team achieved this by exploiting the concept of ynthetic lethality where a combination of two factors kills a cancer cell.
This has the potential to be a less toxic and more effective treatment than more standard approaches because it can specifically target cancer cells.
Importantly, the research team, funded by Cancer Research UK and the Medical Research Council, have developed also a biomarker test to identify SETD2 mutated tumours,
something that can be used immediately in cancer diagnosis. Professor Tim Maughan, Clinical Director of the Cancer Research UK/Medical Research Council Oxford Institute for Radiation Oncology, said:
his novel and exciting finding provides a new scientific basis for precision targeting of some cancers
which are currently very difficult to treat, and we are now taking these findings into clinical trials.
the hope is that these findings will help to target other cancers with similar weak points
and provide a step towards personalized cancer therapy i
#Google Project Loon Set to Enmesh the Globe with Internet Balloons by 2016 Project Loon is yet another highly ambitious project of the tech-giant Google,
#Study reveals structure of tuberculosis enzyme, could offer drug target A team of scientists, including several from the U s. Department of energy Argonne National Laboratory,
have determined the structures of several important tuberculosis enzymes, which could lead to new drugs for the disease.
Tuberculosis, caused by Mycobacterium tuberculosis bacteria, has proved incredibly stubborn even in the age of powerful antibiotics,
infecting about one third of all people worldwide. Treatment can take up to nine months. It has stealth properties that protect it from antibiotics;
said Andrzej Joachimiak, an Argonne Distinguished Fellow, head of the Structural biology Center, co-principal investigator at the Center for Structural genomics of Infectious diseases and a corresponding author on the new study.
It so essential that virtually all living organisms, including human and bacterial pathogens, have versions of it. hat we discovered earlier this year is that the human and bacterial versions bind molecules differently,
Joachimiak said. his is very important for finding a molecule to build a drug aroundou don want to inhibit a human enzyme, just the pathogen one.
Helena Boshoff at the National Institute of Allergies and Infectious diseases performed complementary studies showing that these inhibitors do in fact efficiently block mycobacterium growth l
For the first time, scientists have identified a protein inside blood vessels found at the invasive edge of brain tumours highlighting the area from where cancer is most likely to spread.
Mapping this inflammation gives scientists a more complete picture of the cancer. The scientists have developed a special dye that recognises
and Cancer Research UK scientist at The University of Oxford, said: f we can map the edge of the tumour,
surgery and radiotherapy often fail to remove aggressive tumour cells and the brain tumour can grow back. his research shows that we can improve imaging of brain tumours,
which could help both surgeons and radiotherapists with more effective treatment. Professor Charlie Swanton, NCRI chair and Cancer Research UK scientist at the Francis Crick Institute, said:
rain tumours are very difficult to treat and take the lives of too many patients each year.
and spread has potential to really help doctors treat patients and help save more lives.
Harpal Kumar, Cancer Research UK chief executive, said: rain cancers continue to have very poor survival rates,
which is why research into how to treat them is a top priority for Cancer Research UK.
Being able to delineate the edges of brain tumours is an exciting step towards better surgery and radiotherapy for patients.
The holy grail would be to be able to completely remove brain tumours with the help of this new imaging technique reducing recurrence of the disease
and saving more lives. i
#Whopping Galaxy cluster Spotted with Help of NASA Telescopes Astronomers have discovered a giant gathering of galaxies in a very remote part of the universe, thanks to NASA Spitzer space telescope and Wide-field Infrared Survey Explorer (WISE.
They also discovered that SWEETS make plants susceptible to hijacking by pathogens that steal plant energy supplies before they can be transported to the seeds.
#Researchers Smash Records with Pig-to-Primate Organ transplants A biotech company is genetically engineering pigs so that their organs might work in people.
With the financial aid of a biotechnology executive whose daughter may need a lung transplant, U s. researchers have been shattering records in xenotransplantation,
or between-species organ transplants. The researchers say they have kept a pig heart alive in a baboon for 945 days
The GM pigs are being produced in Blacksburg, Virginia, by Revivicor, a division of the biotechnology company United Therapeutics.
Rothblatt says her goal is to create n unlimited supply of transplantable organsand to carry out the first successful pig-to-human lung transplant within a few years.
One of her daughters has a usually fatal lung condition called pulmonary arterial hypertension. In addition to GM pigs
Some researchers agree with Rothblatt that the latest results mean pig-to-human transplants are plausible. think it possible;
a Swiss transplant surgeon in Geneva. He said he would transplant a genetically engineered pig organ into a patient today,
In fact, thousands of people die each year while waiting on transplant lists. Donated human organs are scarce,
complete with a surgical theater and a helipad so organs can be whisked where they are needed.
The last time a doctor transplanted a pig heart into a person, in India in 1996,
leading transplant surgeons have been meeting with Revivicor ever few months to plan what genes they like to see added next.
or 100 iterations. et surgeons credit the genetically enhanced pigs with some recent successes. Muhammad Mohiuddin,
a transplant surgeon and researcher at the National Heart, Lung, and Blood Institute, in Bethesda, Maryland, says a heart from one of Revivicor pigs lasted two and a half years inside a baboon.
achieved by Massachusetts General Hospital. Also this summer, transplant experts at the University of Pittsburgh said they kept a baboon alive with one of Revivicor pig kidneys for more than four months.
The heart transplants were not life-sustaining but eterotopic? the pig heart was attached to the baboon circulatory system
when a transplant into humans could occur. That is because surgeons still need to completely replace a baboon heart with one from these pigs
and show it keeps the animal alive. t wouldn be serious to give a time line for use in humans,
Lung transplants will be harder, since lungs are permeated with blood vessels and heavily exposed to the immune system.
So far, transplants last only a matter of days, says Rothblatt. She has been financing research at the University of Maryland,
but lungs are very difficult. ransplant surgeons say one of the largest obstacles they face is the immense cost of carrying out xenotransplant experiments.
A single transplant surgery costs $100, 000 and involves eight people. Then there the cost of keeping the primates, the red tape of animal regulations,
Genome pioneer Craig Venter led a team that manufactured a genome for a germ that causes pneumonia in cows,
says Romesberg. he home run is the ability to produce therapeutic proteins with unnatural amino acids in them. ynthorx achievement falls a bit short of that.
including new vaccines. It might be possible, for instance, to make a tuberculosis virus with unnatural DNA in it.
It would be a real, living virus. But without any raw material to copy its genes (that is,
but also benign, that would be the perfect vaccine, says Schultz. Synthetic life forms have implications far beyond new products.
Schultz thinks there will be medical uses of synthetic organisms long before they are released into the environment to eat oil
And once synthetic biology leads to a new drug or vaccine, he thinks, wel get used to the idea of inventing life for our own good. ne has to pick the most near-term applications of this technology to show what it can really do for the good of mankind,
Schultz says. think medicine is one area of pretty obvious applications. d
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