#Gene therapy Restores Hearing In Deaf Mice Using gene therapy, researchers at Boston Children's Hospital and Harvard Medical school have restored hearing in mice with a genetic form of deafness.
Their work, published online July 8 by the journal Science Translational Medicine, could pave the way for gene therapy in people with hearing loss caused by genetic mutations."
"Our gene therapy protocol is not yet ready for clinical trials--we need to tweak it a bit more
--but in the not-too-distant future we think it could be developed for therapeutic use in humans,
Charles Askew More than 70 different genes are known to cause deafness when mutated. Holt, with first author Charles Askew and colleagues at École Polytechnique Fédérale de Lausanne in Switzerland, focused on a gene called TMC1.
because it is a common cause of genetic deafness, accounting for 4 to 8 percent of cases,
Children with two mutant copies of TMC1 have profound hearing loss from a very young age, usually by around 2 years.
--and is a good model for the dominant form of TMC1-related deafness. In this form, less common than the recessive form, a single copy of the mutation causes children to gradually go deaf beginning around the age of 10 to 15 years.
To deliver the healthy gene, the team inserted it into an engineered virus called adeno-associated virus 1,
In the recessive deafness model, gene therapy with TMC1 restored the ability of sensory hair cells to respond to sound--producing a measurable electrical current--and also restored activity in the auditory portion of the brainstem.
Most importantly, the deaf mice regained their ability to hear. To test hearing, the researchers placed the mice in a"startle box
In the dominant deafness model, gene therapy with a related gene, TMC2, was successful at the cellular and brain level,
and is already in use in human gene therapy trials for blindness, heart disease, muscular dystrophy and other conditions.
"Current therapies for profound hearing loss like that caused by the recessive form of TMC1 are hearing aids,
and cochlear implants,"says Margaret Kenna, MD, MPH, a specialist in genetic hearing loss at Boston Children's Hospital who is familiar with the work."
"Cochlear implants are great, but your own hearing is better in terms of range of frequencies, nuance for hearing voices, music and background noise,
and figuring out which direction a sound is coming from. Anything that could stabilize or improve native hearing at an early age is really exciting
"Holt believes that other forms of genetic deafness may also be amenable to the same gene therapy strategy.
Overall, severe to profound hearing loss in both ears affects 1 to 3 per 1, 000 live births.""I can envision patients with deafness having their genome sequenced and a tailored,
precision medicine treatment injected into their ears to restore hearing, "Holt says. Sound transducers: How TMC works Holt's team showed in 2013 that TMC1
and the related protein TMC2 are critical for hearing, ending a rigorous 30-year search by scientists.
a mutation in the TMC1 gene is sufficient to cause deafness. However, Holt's study also showed that gene therapy with TMC2 could compensate for loss of a functional TMC1 gene,
restoring hearing in the recessive deafness model and partial hearing in the dominant deafness model."
"This is a great example of how the basic science can lead to clinical therapies, "says Holt."
and can ultimately challenge, the burden of deafness in humans. The results are testament to the immense dedication of the research team
It was observed by a team of researchers including Gilles Hickson, an assistant professor at the University of Montreal Department of Pathology and Cell biology and researcher at the CHU Sainte-Justine Research Centre, his assistant Silvana Jananji, in collaboration with Nelio
it can be a source for triggering cancer, for example, said Hickson. It is well known that microscopic cable-like structures,
and to certain diseases, said Hickson, who has devoted the last 15 years of his research life to cell biology.
In fact, all cancers are unchecked characterised by cell division, and the underpinning processes are potential targets for therapeutic interventions that prevent cancer onset
and spread. ut before we get there, we must continue to expand our knowledge about the basic processes
Ultimately, this could help the rational design of more specific therapies to inhibit the division of cancer cells,
actually gives impulses through the brain into the muscles, then the muscles contract,"orthopaedic surgeon and director of research and development at Ossur,
Invented by an optometrist in Canada, the Ocumetics Bionic Lens promises to enhance eyesight to a level that's three times better than 20/20-the universal standard for normal vision.
though-the lens developed by Gareth Webb is inserted into the eye via a painless procedure that takes less than 10 minutes (Webb says the process is a lot like cataract surgery).
so you'll never have a problem with cataracts or failing vision no matter how long you live.
dedicated to helping organisations that provide eye surgery in developing countries. Funds have also been earmarked for eye research institutes across the world d
The idea came to University of Guelph public health researcher Christopher Charles, during a trip to Cambodia six years ago,
000 fish have been distributed to hospitals and charity organisations around the country. Being deficient in iron means the body doesn produce enough red blood cells,
making us tired, pale and more vulnerable to illness. It hits women and children the hardest, with almost 50 percent in the developing world being diagnosed with anaemia.
and the disorder can be caused by a multitude of things, such as vitamin deficiencies and parasitic infections.
and it become one of the biggest challenges facing public health today. But new findings suggest that old,
More recently, researchers at St jude Children Research Hospital in Memphis revisited the drug, with an interest in increasing its potency.
it could more easily bind to the cell ribosomes to fight a range of bacterial infections. his study demonstrates how classic antibiotics derived from natural products can be redesigned to create semisynthetic compounds to overcome drug resistance, one of the team,
as well as respiratory tract infections, including pneumonia and influenza. Publishing the results in Science Translational Medicine, the team observed no serious side effects.
Studies on mice and rats showed the drug could also be used to prolong survival in particularly severe pneumonia,
while previous research published last year in Nature Medicine has shown this class of drugs could also be effective in treating tuberculosis.
The particular variety-known as 1599-appears to be just as effective as current TB drugs on the market,
"I hope the result will be drugs that are more effective against tuberculosis and offer a faster route to a cure with fewer side effects,"said Lee.
With TB remaining a leading cause of global illness and death, killing roughly 1. 3 million people each year,
we certainly hope so too i
#Drug based on herpes successfully treats skin cancer patients A modified version of the herpes virus has been used to treat skin cancer patients,
and one in four have responded positively to the treatment, remaining in remission at least six months afterwards.
The results come from a clinical trial in the UK involving more than 400 patients with aggressive melanoma,
who signed up to be treated through virotherapy-a technique that uses altered viruses to attack specific pathogens such as cancer cells."
from the Institute of Cancer Research London, told Hannah Devlin at The Guardian. The herpes-based drug, named Talimogene Laherparepvec (T-VEC), has proven so effective,
Harrington and his team hope to see it on the market by 2016. A successful phase 3 trial means the only remaining hurdle for it to be sold commercially in the US and Europe by pharmaceuticals company,
Amgen, is to get approval from the FDA and the European Medicines Agency. The drug is administered once every two weeks for up to 18 months
and while participants in the trial received flu-like side effects after the first few injections, this was far preferable to the side effects that come with chemotherapy drugs.
According to the study, published in The Journal of Clinical Oncology, of the 436 patients with inoperable melanoma, 16.3 percent of them were still in remission six months after the treatment,
which boosts a patient immune response to the cancer. Of those who took the T-VEC drug,
'having showed no signs of the cancer following the treatment, and during the trial, the T-VEC group lived an average of 41 months,
or metastatic form of the cancer, were too far along to respond to conventional treatments,
even if the cancer had spread to various other organs in the body.""They had ranged disease that from dozens to hundreds of deposits of melanoma on a limb all the way to patients where cancer had spread to the lungs and liver,
"Harrington told her. Harrington, who has been developing the treatment for over 10 years, has been trying it out across a range of different cancer types,
and found that melanoma responded the most positively to it. The team figured out how to harness its amazing capacity for replication by removing two genes that made it impossible for it to multiply inside healthy cells,
but it still had its run of things inside the cancerous melanoma cells.""Meanwhile, T-VEC has also been modified to produce a molecule called GM-CSF,
which serves as a red flag waved at the immune system,"Rachel Feltman reports for The Washington post."
"So in addition to the destructive power of the T-VEC cells themselves, the therapy summons the immune system right to where it's needed-the tumour."
"The team will continue to test out the drug on other forms of cancer through clinical trials,
how cancer begins to spread through the body o
#Engineers have created a computer that operates on water droplets Researchers in the US have built a fully functioning computer that runs like clockwork-but instead of electrons,
#Injectable electronics now exist that could one day help treat paralysis It sounds like something taken straight from a science-fiction movie,
and his colleagues could lead to new ways to treat neurodegenerative diseases and paralysis, as well as mapping out the brain in greater detail than ever before.
#These tiny plastic chips can deliver therapeutic genes into cells A graduate student is developing a cost-effective new method of delivering desirable genes into human cells using a tiny plastic chip.
and extend the reach of new cancer-killing immunotherapy treatments, which currently rely on viral vectors and cost around $5, 000-$10, 000 a pop.
Lab-on-a-chip devices are already being used around the world to help provide on-the-spot diagnoses for diseases such as HIV and Ebola,
and reprogram them to have a therapeutic effect. his process could substantially bring down the cost of gene therapy,
and turning on genes inside them to activate them against certain tumours or diseases, before then inserting these cells back into the patient.
it has far fewer side effects than traditional therapies, and early trials show that they may also be far more effective-with studies so far proving immunotherapy is successful against aggressive forms of melanoma and glioblastoma.
But the problem is, in order to turn on genes against the desired disease, scientists need to deliver DNA into the patient cells,
and right now our best way of doing this is to use viral vectors, which are expensive,
Manufacturing these therapies by viral vectors requires industrial-sized manufacturing plants and costs tens of millions of dollars but it only enables the treatment of thousands of patients per year.
Even better, theye also been able to show that the therapeutic cells their lab-on-a-chip creates are highly viable
otherwise as a result of a bone transplant. The drug hasn been tested on people just yet, but the team are now working on developing it for human use within the next three years,
and improve survival outcomes after surgery and injury.""We are excited very,"lead researcher Sanford Markowitz,
an oncologist from Case Western Reserve University in the US, said in a press release.""We have developed a drug that acts like a vitamin for tissue stem cells,
or are about to undergo bone marrow transplants or liver surgery but in the future they hope to see
whether it could more broadly speed up the repair of tissues around the body.""The drug heals damage in multiple tissues,
which suggests to us that it may have applications in treating many diseases, "said Markowitz. The drug,
In fact, mice given SW033291 recovered normal blood counts after bone marrow transplants six days faster than mice that weren treated.
The drug also almost completely healed mice with ulcers in their colon, and helped mice that had had parts of their liver removed regrow the tissue nearly twice as fast as the control group.
Most impressively, the team tested out the drug on mice that had received lethal doses of radiation before being given a partial bone marrow transplant.
#A new smartphone attachment could save you a trip to the eye doctor You might think nothing of a trip to the optometrist,
The World Health Organisation (WHO) estimates that 80 percent of visual impairments suffered worldwide could be prevented
you can essentially carry an optometrist's fundus camera around in your back pocket.""Conduct routine eye examinations and retinal screenings anywhere for possible detection of a variety of disorders, including the leading causes of blindness-cataracts, glaucoma, diabetic retinopathy and age-related macular degeneration,
"the inventors explain on the project's official site. The gadget has been developed by ophthalmologist Andrea Russo together with and Italian tech development firm Si14 Spa.
Once the link between doctor and patient is established, doctors are able to remotely control the device to choose where in the eye to look,
as well as set and review patient files. It provides a comprehensive view of the inside of the eyeball (including the retina
optic disc, macula, fovea and posterior pole. As Digital Trends reports, the D-EYE uses a combination of beam splitters
It can compensate between-10d myopia (nearsightedness) to+10d hyperopia (farsightedness. At the moment the lens costs#400 (AUD$800) with the bumper attachment selling individually for#40 (AUD$80),
and get the results back to a professional optometrist-the idea is that the expert eye doctor can see more patients more quickly without leaving the office or waiting for appointments.
and get it into the hands of people who can't make it to the optometrist clinic-that could be
because they have a disability of some kind or because they live a long way from the nearest healthcare centre e
and stop epidemics One potential use for drones that you might not have thought about is preventing the spread of disease.
In remote areas where dengue fever or malaria can take hold, the impact of drone technology and number of saved lives could be huge.
and analysing the diseases they're carrying:""The mosquito is the most dangerous animal on the planet,
because it carries so many pathogens,"Microsoft researcher Ethan Jackson, who is leading Project Premonition, told Allison Linn over on the company's blog."
This would allow scientists to not only monitor the spread of known diseases carried by mosquitoes,
and epidemics before they begin to spread. To do this, they're developing software that will be able to quickly
Getting advance warning of a potential epidemic is crucial in stopping or limiting it. Vaccines and health clinics can be up and running earlier
and any necessary travelling restrictions can be put in place before the situation worsens.""The ability to predict an epidemic would be huge,"Douglas Norris,
a professor of molecular microbiology and immunology at Johns Hopkins Bloomberg School of Public health in Maryland, told Linn.
As part of his work, Norris often finds himself working in remote areas using mosquito traps that haven't changed much since the 1950s or 60s.
#Autonomous robot arms are going to 3d-print a bridge in Amsterdam From low-cost housing to lifesaving implants,
it does offer some really interesting potential for people with disabilities. Tim Mildon from the BBC predicted that in the next 10 or 20 years,
the technology will be ready to assist people with severe disabilities and limited movement.""It is part of the BBC's research into how technology might make its services more accessible to people with disabilities,"Stuart Dredge reports at The Guardian.
Gotta start somewhere, right? The future of television is millions of people simultaneously rage quiting Game of Thrones with their minds every time religion burns a child i
The scientists are now looking for ways they can use the same approach to develop more effective and less toxic cancer treatments in humans.
and suggest future avenues for developing effective cancer treatments. ost of the drugs we use to fight cancer are designed to kill cancerous cells.
and the side effects can be intense. reatment regimes for advanced colorectal cancer involve combination chemotherapies that are toxic and largely ineffective,
yet have remained the backbone of therapy over the last decade, "said senior researcher Scott Lowe from the Weill Cornell Medical College in New york
. But his team may have now found a new way to fight the cancer type, by reactivating a gene known as adenomatous polyposis coli (Apc) that turned off in 90 percent of human colorectal tumours.
and six months later there were no signs of the cancer coming back. While scientists have looked previously into turning certain genes on or off in animal models in order to fight cancer,
they've struggled to do so without triggering excess gene activity and causing other problems in normal cells.
Even more impressive was the fact that this approach worked on mice with malignant colorectal cancer tumours that contain Kras
"It is currently impractical to directly restore Apc function in patients with colorectal cancer, and past evidence suggests that completely blocking Wnt signaling would likely be severely toxic to normal intestinal cells,
whether Wnt inhibition or similar approaches would provide long-term therapeutic value in the clinic.""And while this specific treatment will most likely only work for colon cancer,
the team believes that the same approach could be tweaked to suit other cancer types.""If we can define which types of mutations
we will be equipped better to identify the most appropriate treatments for individual cancers, said Lukas Dow, one of the researchers.
and a microwave Carbon nanoparticles can be incredibly useful in the treatment of many types of disease,
and deliver medicine to wherever it's most needed in the body. They're also relatively easy to track as they move through the system,
and interfere with them as they deliver their medicine.""These tiny particles are camouflaged kind of, I would say,
"That clinical use involves the carbon spheres being coated with polymer-a polymer that can gradually release drugs into the system to fight cancer and other diseases.
"This is a versatile platform to carry a multitude of drugs-for melanoma, for other kinds of cancers and for other diseases,"says Rohit Bhargava."
"You can coat it with different polymers to give it a different optical response. You can load it with two drugs,
so you can do multidrug therapy with the same particles.""H/T: Techrada d
#This new insulin patch could soon replace injections for diabetics A new'smart patch'lined with painless microneedles full of insulin has been developed by researchers in the US in an effort to do away with the uncomfortable injections that have become a part of life for the millions
of type 1 diabetics around the world who need to manually regulate their hormone levels.
The 4-cm patch features more than a 100 of these eyelash-sized microneedles, and not only go they contain enough insulin to provide the same dosage as a single injection,
but they also contain glucose-sensing enzymes that can identify when blood-sugar levels are too high and release the insulin into the blood stream."
"We have designed a patch for diabetes that works fast, is easy to use, and is made from nontoxic,
"The whole system can be personalised to account for a diabetic's weight and sensitivity to insulin,
it was able to regulate the insulin levels of diabetic mice for nine hours straight.
And obviously mice are useless at monitoring their blood-sugar levels the way human diabetics have to right now,
This doesn just mean the patch will be a hell of a lot more convenient for its users than the injection system,
"Injecting the wrong amount of medication can lead to significant complications like blindness and limb amputations,
or even more disastrous consequences such as diabetic comas and death.""The system inside the patch was built to mimic the body own insulin generators, known as beta cells.
Genius."The hard part of diabetes care is not the insulin shots, or the blood sugar checks,
it also reliably restores vision in those who suffer from the degenerative eye disease retinitis pigmentosa.
as the device works in a similar way to the cochlear implant-or'bionic ear'-which has helped restore hearing to hundreds of thousands of people."
"This study shows that the Argus II system is a viable treatment option for people profoundly blind due to retinitis pigmentosa,
"lead researcher Allen C. Ho from the Wills Eye Hospital in Pennsylvania, US, said in a press release."
"The device was created by medical company, Second sight, and works through an electronic device that's implanted onto a patient's retina-the layer of light-sensing cells at the back of the eye.
These signals are beamed wirelessly to the patient's implant, which stimulates the retinal cells with painless electrical pulses.
They worked with 30 patients aged between 28 and 77 who had little or no light perception in both eyes as a result of retinitis pigmentosa.
and only 11 adverse events, most of which arose shortly after surgery and were treated successfully. Only one device had to be removed after it became damaged
which have been published in the journal Ophthalmology, will allow them to begin testing the device in an even broader range of subjects,
including treatment for other diseases and eye injuries,"said Ho. We can't wait t
#Paralysed people have learnt to control robots remotely with their thoughts Scientists have developed technology that's given paralysed people the ability control a robot remotely using just their thoughts,
"Each of the nine subjects with disabilities managed to remotely control the robot with ease after less than 10 days of training,"lead researcher José del R. Millán,
and develop new treatments for neurological disorders, such as spinal cord injuries and Parkinson disease.""Our artificial neuron is made of conductive polymers
So imagine being able to use the device to restore function to paralysed patients, or heal brain damage."
#Abnormal IVF embryos can now be predicted within 30 hours of development The chromosomal abnormalities that affect at least half of the human embryos created for in vitro fertilisation (IVF) can now be predicted within the first 30 hours of development at the cell
The reason such low odds exist is that between 50 and 80 percent of all embryos created for IVF end up developing a severe chromosomal abnormality that can dramatically increase their chances of being miscarried,
This means that abnormalities can be detected at the earliest stage of human development, which will allow doctors to make a more informed choice
when selecting embryos for implantation, and spend less time culturing embryos that were never going to make it.
Prior to the discovery, abnormalities could only be detected at day five or six which means these embryos have to be cultured
and a waste of resources for the doctors, who could have been cultivating healthier embryos for her."
the researchers are keen to have continued their research by other teams to figure out even stronger markers for abnormalities i
#Scientists are figuring out how to make medicinal marijuana, without the high Scientists have figured out how to separate the pain relieving qualities of medical marijuana from its psychological side-effects in an effort to offer people a new high-free option.
which is not only responsible for the high associated with the drug-plus hallucinations, delusions, memory loss, and feelings of anxiety or calm-it also been shown to slow tumour growth in mice.
And now, scientists have figured out that the mechanisms by which the drug delivers its desired medical effects
"There has been a great deal of medical interest in understanding the molecular mechanisms at work in THC, so that the beneficial effects can be harnessed without the side-effects,"one of the team,
the researchers figured out that negative psychological side-effects of the drug, such as memory defects, mood swings, anxiety,
and paranoia, were triggered by a single pathway in the brain that was separate from the pathway that triggers the drug cancer-killing properties.
In late-stage patients who had received already surgery, the blood test could also predict their chances of survival and relapses.
symptoms don often show until the disease has progressed to life-threatening stages, which means more than 80 percent of patients end up dying from it."
surgical oncologist David Linehan from the University of Rochester Medical centre in the US told Elaine Schattner at Forbes. The key to the new blood test is a tiny,
And more recently, theye been identified as potential markers for disease. Linehan and his team analysed exosomes in blood samples taken from 190 patients with pancreatic cancer, 32 with breast cancer,
called GPC1, was only being carried by exosomes in the blood of the cancer patients. According to Schattner at Forbes
and how likely it was to progress after surgery, based on how much of the protein was being carried by the exosomes.
because it's one of the cancers we don't have any reliable screening test for one of the researchers,
Derek Raghavan from the Levine Cancer Institute in the US told Linda Carroll at NBC News."It kills people
which means the team still needs to figure out how to properly differentiate between the different types of cancer it can detect in the final analysis.
But one of the team, Raghu Kalluri from the MD Anderson Cancer Centre in the US, told NBC News that he thinks the blood test could be available in as little as a year.
and treating this disease, and fast n
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