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Scientists have found a new way to overcome one of the biggest obstacles to the use of viruses for therapeutic genes.
Scientists from the Institute for research at national level have found a way to overcome one of the biggest obstacles to the use of viruses for administration of therapeutic genes, that is, how to prevent the immune system to neutralize the virus before it has delivered its genetic set.
Gene therapy is one of the most promising possibilities for the treatment of genetic disorders such as muscular dystrophy, congenital blindness and hemophilia. Scientists explore gene therapy as a cure for certain types of cancer, neurodegenerative diseases, viral infections and other acquired diseases. In order to obtain a therapeutic gene into cells, scientists use viruses that deliver its genetic material in cells as part of their normal replication process.
Again and again, these efforts were thwarted by the very immune system of the body that damages each viral vector. Thus the therapeutic gene can be delivered to diseased cells and disease raging in full force.
A team led by Louis-Rodino Klapak, PhD, and Jerry Mendel, MD, principal investigator in the Center for Gene Therapy at Nationwide, show for the first time that using a process called plasmapheresis, just before delivery of the virus for gene therapy he is protected long enough to enter the cell and deliver their genetic material.
In a study of gene therapy for the treatment of Duchenne muscular dystrophy (DMD), Dr. Rodino-Klapak using plasmapheresis in a large animal model, and then injected a virus carrying the gene micro-dystrophin. When studying the level of gene expression of the micro-dystrophin in animals, it was found that there is a 500% increase in the gene expression in the animals who received a plasmapheresis.
Dr. Mendel believes that right now, gene therapy seems to work best in patients who have antibodies to the virus. It is this virus is used to supply the necessary gene. It is exactly this gene, which is a therapeutic, curative intent of the organism suffering from a disease. On the other hand, it limits the number of patients who can benefit from gene therapy. This is because in very few patients lacking antibodies against the virus.
Using the method of plasmapheresis repeatedly increases the potential of gene therapy, as this eliminates the obstacle called immune response of the organism.
As gene therapy becomes more widespread, it may be necessary, patients receiving more than one course of treatment.
The main problem is that when you go home after the first treatment, their body develops antibodies to the virus used to deliver the therapeutic gene. The use of plasma in a patient who previously received gene therapy may allow him to be treated again.
Human stem cells taken from an embryo or fetus delay the development of fatal brain and nerve diseases in mice, researchers reported.
The study was published in the journal Nature Medicine and is the first study in which human embryonic stem cells have been successfully used for the treatment of diseases in animals.
Dr. Evan Snyder of Medicine University of California, head of the study, reported that his team hopes these tests soon be moved on children suffering from fatal and incurable brain diseases such as Alzheimer's Sandhoff.
The approach used in this study could lead to new treatments for neurodegenerative diseases such as Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis and others.
In the study, researchers used mice having disabilities equivalent disability in Sandhoff disease.
Children with this disease have severe mental retardation and motor dysfunction. Death usually occurs in them in childhood. This disease is caused as a result of a mutation in the gene responsible for synthesis of the enzyme hexosaminidase or hexyl, of which the brain needs to purify from the excess of lipids. In accumulation of lipids, brain cells die. The disease is characterized by inflammation that kills brain cells.
Snyder's team used human embryonic stem cells and fetal human stem cells. These cells were transplanted into the brains of mice.
Side effects of treatment were observed, and the mice did not reject the foreign cells and the inflammation is reduced.
Thus treated mice lived 70% longer compared to untreated.
Stem cells are valuable because they can develop into all types of cells and tissues.
Transplanted human cells replace damaged nerve cells and carry nerve signals in the brain. They have also led to an increased synthesis of the enzyme hex, which is insufficient in Sandhoff disease.
Discovered a new molecule - the first of its kind that allows multiplication of stem cells from umbilical cord. This discovery is very important as these cells are used for transplantation for the treatment of blood diseases such as leukemia, lymphoma and myeloma.
The blood from the umbilical cord of the infant is considered an excellent source of hematopoietic stem cells that could be used for transplants.
These stem cells are less likely to cause an adverse reaction by the immunity in the recipient thereof to whom they are transplanted.
Furthermore, unlike the adult stem cells from bone marrow, it is not necessary, and the donor recipient has to have immunological compatibility for successful transplantation.
Such type of treatment, however, can not always lead to successful healing in adults, since the number of the collected stem cells from umbilical cord blood is small and insufficient. Scientists believe that by using the new molecule will be possible multiplication of stem cells to a sufficient quantity for the conduct of successful treatment.
Molecule is called the UM171 and was discovered by researchers from the Institute of Cancer Research and immunological and the University of Montreal, Canada.
Doctrine argue that UM171 has the potential to increase the number of blood units, ready for transplant to 10 times.
Macular degeneration is the leading cause of blindness worldwide. It affects one in five people 75 years of age.
The loss of vision is closely related to the aging process. The infringement is not seen so much as an actual condition, but rather as an inevitable part of aging.
New discovery by scientists from the UK, however, may soon change this data.
Researchers from the University of Southampton, UK, identified a unique type of stem cells in the eye that can be converted into photovoltaic cells. What is more important is that have the potential to turn the processes associated with blindness due to macular degeneration.
Affected by the condition suffer from blurred and "distorted" vision before it completely disappears. To a large extent the cause lies in the loss of photoreceptor cells - cells that require light. In this study, the team of researchers found a way to replace these lost cells and reverse the effects of the condition.
Right on the front surface of the eye - between the cornea and sclera, is an area where stem cells have been shown to "behave" as photoreceptor when they are in a suitable environment.
For the moment, this environment can be created only in the laboratory. Since stem cells exist in the eyes throughout life, it is believed that with more research in this area, they can be used in future therapies for the treatment of blindness caused by macular degeneration.
Enzymes are proteins which accelerate the chemical reaction in the cell. A special portion of the enzyme which is called "active side" is formed in such a way as to fit with the specific substrate molecules.
The enzyme works by binding to one or more specific molecules called "substrate".
Connection occurs on the active side. The enzyme and substrate form a complex enzyme substraten.
The interaction between the enzyme and the substrate either increases or decreases any of the chemical bonds in the substrate.
As a result of the chemical interactions in the active part, to form a new product.
He then released from the active start of the enzyme acquires its normal shape and is ready to work again.
Two Americans have been successfully cured of insidious infection with Ebola, says American media. Yesterday the hospital in Atlanta is written 33-year-old Dr. Kent harridan who was adopted there treatment after his stay in West Africa and insidious virus infection against which to date medicine lacked any effective therapy.
Physician Dr. Bruce Ribnar, infectious diseases specialist from Emory University Hospital informed the Center for Disease Control that the patient is successfully treated and can return to his family and his community without endangering public health.
Days ago was discharged and another patient who has entered into contact with the virus during missionary activity.
Experts say that when they found no evidence for the presence of virus in their body.
At a special press conference Dr. harridan thanked especially the six doctors and 21 nurses for care during treatment in the hospital, and all that he had help to get experimental treatment and the thousands of others who are his courage.
Dr. harridan is infected with dangerous virus while working in Liberia, where he was sent as part of a team of hospital ELWA, medical care in distress by an epidemic of Ebola area. The same body which was sent to Dr. harridan, there is another 23 young doctors.
Infektsionistat Dr. Ribnar seek treatment for infection with Ebola forty years. According to him, it is unlikely that the young doctor falls ill again from the same strain of the virus in any of its meetings, as his body has developed immunity against it, but should not overlook the fact that there are other insidious strains of virus.
Dr. Ribnar said that the treatment used two experimental medicines but still can not decide about their effect, as this is one the first people on which they are tested. This treatment were subjected to three Liberian doctors reported an improvement in the condition. Adult, however, where the drug was applied ZMapp, has died.
The doctor advised when treating patients with Ebola to be approached individually. For recovered can be considered, patients whose blood tests showed no presence of the virus, at least 2-3 days after resolution of symptoms of the disease.
According to him, in patients may experience complete recovery, provided that no organ damage.
A happy cure have yet to recover their strength, while Dr. Ribnar plans to share the experience of the team in the management of dangerous disease. Some of this information in his words will be presented in specialized medical journals. First, however, new knowledge will get doctors who are trying to keep the epidemic of Ebola.
Swiss scientists have developed a new protein structure, which may improve the efficacy of drugs. New synthetic amino acid can be used to create entirely new drugs.
Development of new and effective drugs is a feat for many researchers. Until now, most drugs based on the basis of amino acid obtained naturally. These include insulin hormone, vancomycin and the like.
There are a variety of peptides and proteins, but only 20 different amino acids. They form a unique structure when they are "stacked" together. The synthetic structure created by the Swiss researcher is similar to that of cysteine - sulfuric amino acid. Synthetic however is significantly more efficient.
Disease attack appear more and more people. It takes more effective drugs that can handle the increasing resistance. Like the natural chemical structures may create such a new and improved treatment of various diseases.
Cysteine can form a bridge to the cysteine amino acid found in it, thereby creating 3D structure. The new amino acid, however, can form two bridges, when coupled with other similar amino acid cysteine.
Laboratory tests revealed that the newly synthesized amino acid is eight times more efficient and fight disease by 40-fold higher speed in comparison with a naturally occurring amino acid cysteine.
Treatment of various diseases of the blood and immune system is about to be discovered by scientists that reveal the mystery of the origin of stem cells.
Avstraliyskoro study was conducted by the Institute for Regenerative Medicine at Monash University, Melbourne. It first establishes a mechanism that "unlocks" the body in the formation of hematopoietic stem cells (HSC).
These cells are found in bone marrow and umbilical cord and are extremely important because they can "feed" the stock of blood cells in the body. Patients with leukemia have been successfully treated with HSC transplantation. Medical experts, however, believe that they can come into wider use.
According to the lead researcher - Prof. Peter Curry, understanding the way the HSC is samopodnovyavat to fill blood cells is the "Holy Grail" of stem cell biology.
Hematopoietic stem cells are the best therapeutic agent, since they can produce any cell in the body, said Curry, adding that they could be used for the treatment of severe blood diseases. But before that you need to understand how they emerge.
During this study, researchers observed cells in the developing zebra fish - tropical freshwater fish known for its regenerative abilities and optically visible embryos.
Using a microscope with high resolution scientists capture the manner in which the cells grow. The process of their formation is captured in dramatic detail.
Observations found that HSC need a "friendly" type of cell that help their formation. Metaphorically, the researchers explain that these cells serve as a comfortable sofa where HSC accommodate. Researchers establish whether the genes that are required for the formation of these "friendly" cells.
The most remarkable achievement of the experts is that they can identify the signals in these cells, helper, which are responsible for the formation of HSC, after which they could be used in vitro for the creation of various blood cells to "fix" all types of diseases related to the blood.
Scientists have discovered a previously unknown virus that lives in the human gut, according to a study published in Nature Communications.
An international team of scientists discovered the virus in CrAssphage genetic material from samples of intestine. Scientists believe the virus can affect the behavior of some of the most common bacteria in the intestinal flora.
Experts explain that these types of viruses called bacteriophages play an important role in chronic diseases. Leading a team from the State University at San Diego, USA, "clears" the genetic information that is stored in three large international databases.
They fall on the portion of the DNA to be detected in more than half of all the samples. Further analysis found that the virus has not been detected so far.
Scientists explain that it has a genetic fingerprint of the bacteriophage - type virus, which is known to infect the bacteria. He can control the behavior of bacteria that affect - in some cases makes it easier to live in the environment in which they are located, and may make the bacteria more powerful.
Scientists are trying to grow the virus in the laboratory. Explain that the next step will be to establish exactly the way in which the virus affects intestinal bacteria.
Fungal molecule allows to realize the effect of the antibiotic to achieve the cure. It can be the key to suppression of resistance to antibiotics, according to scientists from the University of McMaster, Canada.
The emergence of antibiotic resistance, role playing gene known as NDM-1. The World Health Organization recognizes it as a global threat to human health due to its resistance to some of the most powerful class of antibiotics known as carbapenems.
Despite his frightening presence in many bacterial strains, NDM-1 is found in the most common bacteria among people - Escherichia coli - bacteria that causes most infections of the bladder and kidneys. Without antibiotics to "fight" with NDM-1, doctors are helpless.
The carbapenem class of drugs are very similar to penicillin, which means that they are used in a variety of unique situations. Researchers worse is that NDM-1 affects many organisms which themselves cause all sorts of "challenging" conditions and now have multi-drug resistance.
Before fungal molecule to be detected in the soil in Nova Scotia, Canada, researchers found that NDM-1 requires zinc to "grow." The challenge in them was to find a way to remove zinc safely and without causing the appearance of side effects for the patient. With the discovery of the molecule, known as AMA, this extraction becomes possible.
During the laboratory studies, the experts found that it is really effective and allows the antibiotic to act in order to achieve cure. But there is still much work to researchers, pending the development of a "global" solution.