This article is about the medical aspects of bone marrow in humans. For use of animal marrow in cuisine, see Bone marrow (food).

Bone marrow is the flexible tissue in the interior of bones. In humans, red blood cells are produced in the heads of long bones in a process known as hematopoiesis. On average, bone marrow constitutes 4% of the total body mass of humans; in an adult weighing 65 kilograms (143lb), bone marrow accounts for approximately 2.6 kilograms (5.7lb). The hematopoietic component of bone marrow produces approximately 500 billion blood cells per day, which use the bone marrow vasculature as a conduit to the body's systemic circulation.[1] Bone marrow is also a key component of the lymphatic system, producing the lymphocytes that support the body's immune system.[2]

Bone marrow transplants can be conducted to treat severe diseases of the bone marrow, including certain forms of cancer. Additionally, bone marrow stem cells have been successfully transformed into functional neural cells,[3] and can also potentially be used to treat illnesses such as inflammatory bowel disease[4] and, in some cases, HIV.[5][6]

The two types of bone marrow are medulla ossium rubra (red marrow), which consists mainly of hematopoietic tissue, and medulla ossium flava (yellow marrow), which is mainly made up of fat cells. Red blood cells, platelets and most white blood cells arise in red marrow. Both types of bone marrow contain numerous blood vessels and capillaries. At birth, all bone marrow is red. With age, more and more of it is converted to the yellow type; only around half of adult bone marrow is red. Red marrow is found mainly in the flat bones, such as the pelvis, sternum, cranium, ribs, vertebrae and scapulae, and in the cancellous ("spongy") material at the epiphyseal ends of long bones such as the femur and humerus. Yellow marrow is found in the medullary cavity, the hollow interior of the middle portion of long bones. In cases of severe blood loss, the body can convert yellow marrow back to red marrow to increase blood cell production.

The stroma of the bone marrow is all tissue not directly involved in the marrow's primary function of hematopoiesis. Yellow bone marrow makes up the majority of bone marrow stroma, in addition to smaller concentrations of stromal cells located in the red bone marrow. Though not as active as parenchymal red marrow, stroma is indirectly involved in hematopoiesis, since it provides the hematopoietic microenvironment that facilitates hematopoiesis by the parenchymal cells. For instance, they generate colony stimulating factors, which have a significant effect on hematopoiesis. Cell types that constitute the bone marrow stroma include:

Macrophages contribute especially to red blood cell production, as they deliver iron for hemoglobin production.

The blood vessels of the bone marrow constitute a barrier, inhibiting immature blood cells from leaving the marrow. Only mature blood cells contain the membrane proteins required to attach to and pass the blood vessel endothelium. Hematopoietic stem cells may also cross the bone marrow barrier, and may thus be harvested from blood.

The bone marrow stroma contains mesenchymal stem cells (MSCs),[7] also known as marrow stromal cells. These are multipotent stem cells that can differentiate into a variety of cell types. MSCs have been shown to differentiate, in vitro or in vivo, into osteoblasts, chondrocytes, myocytes, adipocytes and beta-pancreatic islets cells. MSCs can also transdifferentiate into neuronal cells.[3]

In addition, the bone marrow contains hematopoietic stem cells, which give rise to the three classes of blood cells that are found in the circulation: white blood cells (leukocytes), red blood cells (erythrocytes), and platelets (thrombocytes).[7]

Biological compartmentalization is evident within the bone marrow, in that certain cell types tend to aggregate in specific areas. For instance, erythrocytes, macrophages, and their precursors tend to gather around blood vessels, while granulocytes gather at the borders of the bone marrow.

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Bone marrow - Wikipedia, the free encyclopedia

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Induced pluripotent stem cells,[1] commonly abbreviated as iPS cells or iPSCs are a type of pluripotent stem cell artificially derived from a non-pluripotent cell typically an adult somatic cell by inducing a "forced" expression of specific genes.

Induced pluripotent stem cells are similar to natural pluripotent stem cells, such as embryonic stem (ES) cells, in many aspects, such as the expression of certain stem cell genes and proteins, chromatin methylation patterns, doubling time, embryoid body formation, teratoma formation, viable chimera formation, and potency and differentiability, but the full extent of their relation to natural pluripotent stem cells is still being assessed.[2] Induced pluripotent cells have been made from adult stomach, liver, skin cells, blood cells, prostate cells and urinary tract cells.[3]

iPSCs were first produced in 2006 from mouse cells and in 2007 from human cells in a series of experiments by Shinya Yamanaka's team at Kyoto University, Japan, and by James Thomson's team at the University of Wisconsin-Madison. For her iPSC research, Dr. Nancy Bachman, of Oneonta, NY, was awarded the Wolf Prize in Medicine in 2012 (along with John B. Gurdon).[4][5][6] For his iPSC discovery (and for deriving the first human embryonic stem cell), James Thomson received the 2011 Albany Medical Center Prize for Biomedical Research and the 2011 King Faisal International Prize, which he shared with Yamanaka. In October 2012, Yamanaka and fellow stem cell researcher John Gurdon were awarded the Nobel Prize in Physiology or Medicine "for the discovery that mature cells can be reprogrammed to become pluripotent."[7]

iPSCs are an important advance in stem cell research, as they may allow researchers to obtain pluripotent stem cells, which are important in research and potentially have therapeutic uses, without the controversial use of embryos. Because iPSCs are developed from a patient's own somatic cells, it was believed that treatment of iPSCs would avoid any immunogenic responses; however, Zhao et al. have challenged this assumption.[8]

Depending on the methods used, reprogramming of adult cells to obtain iPSCs may pose significant risks that could limit their use in humans. For example, if viruses are used to genomically alter the cells, the expression of cancer-causing genes "oncogenes" may potentially be triggered. In February 2008, scientists announced the discovery of a technique that could remove oncogenes after the induction of pluripotency, thereby increasing the potential use of iPS cells in human diseases.[9] In April 2009, it was demonstrated that generation of iPS cells is possible without any genetic alteration of the adult cell: a repeated treatment of the cells with certain proteins channeled into the cells via poly-arginine anchors was sufficient to induce pluripotency.[10] The acronym given for those iPSCs is piPSCs (protein-induced pluripotent stem cells).

Dedifferentiation to totipotency or pluripotency: an overview of methods. Various methods exist to revert adult somatic cells to pluripotency or totipotency. In the case of totipotency, reprogramming is mediated through a mature metaphase II oocyte as in somatic cell nuclear transfer (Wilmut et al., 1997). Recent work has demonstrated the feasibility of enucleated zygotes or early blastomeres chemically arrested during mitosis, such that nuclear envelope break down occurs, to support reprogramming to totipotency in a process called chromosome transfer (Egli and Eggan, 2010). Direct reprogramming methods support reversion to pluripotency; though, vehicles and biotypes vary considerably in efficiencies (Takahashi and Yamanaka, 2006). Viral-mediated transduction robustly supports dedifferentiation to pluripotency through retroviral or DNA-viral routes but carries the onus of insertional inactivation. Additionally, epigenetic reprogramming by enforced expression of OSKM through DNA routes exists such as plasmid DNA, minicircles, transposons, episomes and DNA mulicistronic construct targeting by homologous recombination has also been demonstrated; however, these methods suffer from the burden to potentially alter the recipient genome by gene insertion (Ho et al., 2010). While protein-mediated transduction supports reprogramming adult cells to pluripotency, the method is cumbersome and requires recombinant protein expression and purification expertise, and reprograms albeit at very low frequencies (Kim et al., 2009). A major obstacle of using RNA for reprogramming is its lability and that single-stranded RNA biotypes trigger innate antiviral defense pathways such as interferon and NF-B-dependent pathways. In vitro transcribed RNA, containing stabilizing modifications such as 5-methylguanosine capping or substituted ribonucleobases, e.g. pseudouracil, is 35-fold more efficient than viral transduction and has the additional benefit of not altering the somatic genome (Warren et al., 2010). An overarching goal of reprogramming methods is to replace genes with small molecules to assist in reprogramming. No cocktail has been identified to completely reprogram adult cells to totipotency or pluripotency, but many examples exist that improve the overall efficiency of the process and can supplant one or more genes by direct reprogramming routes (Feng et al., 2009; Zhu et al., 2010).

iPS cells are typically derived by transfection of certain stem cell-associated genes into non-pluripotent cells, such as adult fibroblasts, although this technique is becoming less popular since it is known to be prone to inducing cancer formation. Transfection is typically achieved through viral vectors, such as retroviruses. Transfected genes include the master transcriptional regulators Oct-3/4 (Pou5f1) and Sox2, although it is suggested that other genes enhance the efficiency of induction. After 34 weeks, small numbers of transfected cells begin to become morphologically and biochemically similar to pluripotent stem cells, and are typically isolated through morphological selection, doubling time, or through a reporter gene and antibiotic selection.

Induced pluripotent stem cells were first generated by Shinya Yamanaka's team at Kyoto University, Japan in 2006. Yamanaka used genes that had been identified as particularly important in embryonic stem cells (ESCs), and used retroviruses to transduce mouse fibroblasts with a selection of those genes. Eventually, four key pluripotency genes essential for the production of pluripotent stem cells were isolated; Oct-3/4, SOX2, c-Myc, and Klf4. Cells were isolated by antibiotic selection of Fbx15+ cells. However, this iPS cell line showed DNA methylation errors compared to original patterns in ESC lines and failed to produce viable chimeras if injected into developing embryos.

In June 2007, the same group published a breakthrough study along with two other independent research groups from Harvard, MIT, and the University of California, Los Angeles, showing successful reprogramming of mouse fibroblasts into iPS cells and even producing viable chimera. These cell lines were also derived from mouse fibroblasts by retroviral mediated reactivation of the same four endogenous pluripotent factors, but the researchers now selected a different marker for detection. Instead of Fbx15, they used Nanog which is an important gene in ESCs. DNA methylation patterns and production of viable chimeras (and thereby contributing to subsequent germ-line production) indicated that Nanog is a major determinant of cellular pluripotency.[11][12][13][14][15]

Unfortunately, two of the four genes used (namely, c-Myc and KLF4) are oncogenic, and 20% of the chimeric mice developed cancer. In a later study, Yamanaka reported that one can create iPSCs even without c-Myc. The process takes longer and is not as efficient, but the resulting chimeras didn't develop cancer.[16]

Link:
Induced pluripotent stem cell - Wikipedia, the free encyclopedia

Recommendation and review posted by Bethany Smith

By Jeanette Jacknin M.D.

Dr Jacknin will be speaking about Cosmaceuticals at the upcoming 17th World Congress on Anti-Aging and Regenerative Medicine in Orlando, Florida, April 23-25, 2009.

Stem cells have recently become a huge buzzword in the skincare world. But what does this really mean? Skincare specialists are not using embryonic stem cells; it is impossible to incorporate live materials into a skincare product. Instead, companies are creating products with specialized peptides and enzymes or plant stem cells which, when applied topically on the surface, help protect the human skin stem cells from damage and deterioration or stimulate the skin's own stem cells. National Stem Cell was one of the few companies who actually incorporated into their skin care an enzyme secreted from human embryonic stem cells, but they are in the process of switching over to use non-embryonic stem cells from which to take the beneficial enzyme.

Stem cells have the remarkable potential to develop into many different cell types in the body. When a stem cell divides, it can remain a stem cell or become another type of cell with a more specialized function, such as a skin cell. There are two types of stem cells, embryonic and adult.

Embryonic stem cells are exogenous in that they are harvested from outside sources, namely, fertilized human eggs. Once harvested, these pluripotent stem cells are grown in cell cultures and manipulated to generate specific cell types so they can be used to treat injury or disease.

Unlike embryonic stem cells, adult or multipotent stem cells are endogenous. They are present within our bodies and serve to maintain and repair the tissues in which they are found. Adult stem cells are found in many organs and tissues, including the skin. In fact, human skin is the largest repository of adult stem cells in the body. Skin stem cells reside in the basal layer of the epidermis where they remain dormant until they are activated by tissue injury or disease. 1

There is controversy surrounding the use of stem cells, as some experts say that any product that claims to affect the growth of stem cells or the replication process is potentially dangerous, as it may lead to out-of-control replication or mutation. Others object to using embryonic stem cells from an ethical point of view. Some researchers believe that the use of stem cell technology for a topical, anti-aging cosmetic trivializes other, more important medical research in this field.

The skin stem cells are found near hair follicles and sweat glands and lie dormant until they "receive" signals from the body to begin the repair mode. In skincare, the use of topical products stimulates the stem cell to split into two types of cells: a new, similar stem cell and a "daughter" cell, which is able to create almost every kind of new cell in a specialized system. This means that the stem cell can receive the message to create proteins, carbohydrates and lipids to help repair fine lines, wrinkles and restore and maintain firmness and elasticity.1

First to the market in Britain in April 2007 and the U.S. was ReVive's Peau Magnifique, priced at a staggering 1,050. Manufacturers claim it uses an enzyme called telomerase to "convert resting adult stem cells to newly-minted skin cells' and 'effectively resets your skin's "ageing clock" by a minimum of five years'. The product claims long-term use 'will result in a generation of new skin cells, firmer skin with a 45 per cent reduction in wrinkles and increased long-term skin clarity'. Peau Magnifique is the latest in a line of products developed by Dr Gregory Bays Brown, a former plastic surgeon.

In the course of his research into healing burns victims, Dr Brown discovered a substance called Epidermal Growth Factor (EGF) that is released in the body when there is an injury, and, when applied to burns or wounds, dramatically accelerates the healing process. He believed the same molecule could be used to regenerate ageing skin and went on to develop ReVive, a skincare range based around it. 2

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CARLSBAD, CA(Marketwire Sep 25, 2012) International Stem Cell Corporation ( OTCQB : ISCO ) (www.internationalstemcell.com) (ISCO or the Company) a California-based biotechnology company, today announced that the United States Patent and Trademark Office (USPTO) has granted the Company a patent for a method of creating pure populations of definitive endoderm, precursor cells to liver and pancreas cells, from human pluripotent stem cells.This patent is a key element of ISCOs metabolic liver disease program and allows the Company to produce the necessary quantities of precursor cells in a more efficient and cost effective manner. The patent, 8,268,621, adds to the Companys growing portfolio of proprietary technologies relating to the development of potential treatments for incurable diseases using human parthenogenetic Stem Cells (hpSC).Human parthenogenetic stem cells are unique pluripotent stem cells that offer the possibility to reduce the cost of health care while avoiding the ethical issues that surround the use of fertilized human embryos.Aside from the Companys current liver disease program, this new patented method can be used as a route to create pancreatic and endocrine cells that could be used in future studies of diabetes and other metabolic disorders. ISCO currently has the largest collection of hpSC including cell lines which immune match the donor, as is the case with induced pluripotent stem cells (iPS), and cell lines which immune-match millions of individuals and potentially reduce tissue rejection issues.The Company is focusing its therapeutic development efforts on three clinical applications where cell and tissue therapy is already proven but where there currently is an insufficient supply of safe and efficacious cells: Parkinsons disease, inherited/metabolic liver diseases and corneal blindness.

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In 2006, Shinya Yamanaka of Kyoto University in Japan was the first to disprove the previous notion that reversible cell differentiation of mammals was impossible. He reprogrammed a fully differentiated mouse cell into a pluripotent stem cell by introducing four genes, Oct-4, SOX2, KLF4, and Myc, into the mouse fibroblast through gene-carrying viruses. With this method, he and his coworkers created induced pluripotent stem cells (iPS cells), the key component in this experiment.[1] Scientists have been able to conduct experiments that show the ability of iPS cells to treat and even cure diseases. In this experiment, tests were run on mice with inherited sickle cell anemia.Skin cells were turned into cells containing genes that transformed the cells into iPS cells. These replaced the diseased sickled cells, curing the test mice. The reprogramming of the pluripotent stem cells in mice was successfully duplicated with human pluripotent stem cells within about a year of the experiment on the mice.

Sickle cell anemia is a disease in which the body produces abnormally shaped red blood cells. Red blood cells are flexible and round, moving easily through the blood vessels. Infected cells are shaped like a crescent or sickle (the namesake of the disease). As a result of this disorder the hemoglobin protein in red blood cells is faulty. Normal hemoglobin bonds to oxygen, then releases it into cells that need it. The blood cell retains its original form and is cycled back to the lungs and re-oxygenated.

Sickle cell hemoglobin, however, after giving up oxygen, cling together and make the red blood cell stiff. The sickle shape also makes it difficult for the red blood cell to navigate arteries and causes blockages.[2] This can cause intense pain and organ damage. The sickled red blood cells are fragile and prone to rupture. When the number of red blood cells decreases from rupture (hemolysis), anemia is the result. Sickle cells also die in 1020 days as opposed to the traditional 120-day lifespan of a normal red blood cell.

Sickle cell anemia is inherited as an autosomal (meaning that the gene is not linked to a sex chromosome) recessive condition.[2] This means that the gene can be passed on from a carrier to his or her children. In order for sickle cell anemia to affect a person, the gene must be inherited from both the mother and the father, so that the child has two recessive sickle cell genes (a homozygous inheritance). People who inherit one sickle cell gene from one parent and one normal gene from the other parent, i.e. heterozygous patients, have a condition called sickle cell trait. Their bodies make both sickle hemoglobin and normal hemoglobin. They may pass the trait on to their children.

The effects of sickle cell anemia vary from person to person. People who have the disease suffer from varying degrees of chronic pain and fatigue. With proper care and treatment, the quality of health of most patients will improve. Doctors have learned a great deal about sickle cell anemia since its discovery in 1979. They know its causes, its effects on the body, and possible treatments for complications. Sickle cell anemia has no widely available cure. A bone marrow transplant is the only treatment method currently recognized to be able to cure the disease, though it does not work for every patient. Finding a donor is difficult and the procedure could potentially do more harm than good. Treatments for sickle cell anemia are generally aimed at avoiding crises, relieving symptoms, and preventing complications. Such treatments may include medications, blood transfusions, and supplemental oxygen.

During the first step of the experiment, skin cells (also known as fibroblasts) were collected from infected test mice and put in a culture. The fibroblasts were reprogrammed by infecting them with retroviruses that contained genes common to embryonic stem cells. These genes were the same four used by Yamanaka (Oct-4, SOX2, KLF4, and Myc) in his earlier study. The investigators were trying to produce cells with the potential to differentiate into any type of cell needed (i.e. pluripotent stem cells). As the experiment continued, the fibroblasts multiplied into identical copies of iPS cells. The cells were then treated to form the mutation needed to reverse the anemia in the mice. This was accomplished by restructuring the DNA containing the defective globin gene into DNA with the normal gene through the process of homologous recombination. The iPS cells then differentiated into blood stem cells, or hematopoietic stem cells. The hematopoietic cells were injected back into the infected mice, where they proliferate and differentiate into normal blood cells, curing the mice of the disease.[3][4][verification needed]

To determine whether the mice were cured from the disease, the scientists checked for the usual symptoms of sickle cell disease. They examined the blood for mean corpuscular volume (MCV) and red cell distribution width (RDW) and urine concentration defects. They also checked for sickled red blood cells. They examined the DNA through gel electrophoresis, checking for bands that display an allele that causes sickling. Compared to the untreated mice with the disease, which they used as a control, the treated animals had marked increases in RBC counts, healthy hemoglobin, and packed cell volume levels.[5]

Researchers examined the urine concentration defect, which results from RBC sickling in renal tubules and consequent reduction in renal medullary blood flow, and the general deteriorated systemic condition reflected by lower body weight and increased breathing.[5] They were able to see that these parts of the body of the mice had healed or improved. This indicated that all hematological and systemic parameters of sickle cell anemia improved substantially and were comparable to those in control mice.[5] They cannot say if this will work in humans because a safe way to inject the genes for the induced pluripotent cells is still needed.[citation needed]

The reprogramming of the induced pluripotent stem cells in mice was successfully duplicated in humans within a year of the successful experiment on the mice. This reprogramming was done in several labs and it was shown that the iPS cells in humans were almost identical to original embryonic stem cells (ES cells) that are responsible for the creation of all structures in a fetus.[1] An important feature of iPS cells is that they can be generated with cells taken from an adult, which would circumvent many of the ethical problems associated with working with ES cells. These iPS cells also have potential in creating and examining new disease models and developing more efficient drug treatments.[6] Another feature of these cells is that they provide researchers with a human cell sample, as opposed to simply using an animal with similar DNA, for drug testing.

One major problem with iPS cells is the way in which the cells are reprogrammed. Using gene-carrying viruses has the potential to cause iPS cells to develop into cancerous cells.[1] Also, an implant made using undifferentiated iPS cells, could cause a teratoma to form. Any implant that is generated from using these iPS cells would only be viable for transplant into the original subject that the cells were taken from. In order for these iPS cells to become viable in therapeutic use, there are still many steps that must be taken.[5][7]

Link:
Induced pluripotent stem cell therapy - Wikipedia, the free ...

Recommendation and review posted by Bethany Smith

Molecular genetics is the field of biology and genetics that studies the structure and function of genes at a molecular level. Molecular genetics employs the methods of genetics and molecular biology to elucidate molecular function and interactions among genes. It is so called to differentiate it from other sub fields of genetics such as ecological genetics and population genetics.

Along with determining the pattern of descendants, molecular genetics helps in understanding developmental biology, genetic mutations that can cause certain types of diseases. Through utilizing the methods of genetics and molecular biology, molecular genetics discovers the reasons why traits are carried on and how and why some may mutate.

One of the first tools available to molecular geneticists is the forward genetic screen. The aim of this technique is to identify mutations that produce a certain phenotype. A mutagen is very often used to accelerate this process. Once mutants have been isolated, the mutated gene can be molecularly identified.

While forward genetic screens are productive, a more straightforward approach is to simply determine the phenotype that results from mutating a given gene. This is called reverse genetics. In some organisms, such as yeast and mice, it is possible to induce the deletion of a particular gene, creating whats known as a gene knockout the laboratory origin of so-called knockout mice for further study. In other words this process involves the creation of transgenic organisms that do not express a gene of interest. Alternative methods of reverse genetic research include the random induction of DNA deletions and subsequent selection for deletions in a gene of interest, as well as the application of RNA interference.

A mutation in a gene can result in a severe medical condition. A protein encoded by a mutated gene may malfunction and cells that rely on the protein might therefore fail to function properly. This can cause problems for specific tissues or organs, or for the entire body. This might manifest through the course of development (like a cleft palate) or as an abnormal response to stimuli (like a peanut allergy). Conditions related to gene mutations are called genetic disorders. One way to fix such a physiological problem is gene therapy. By adding a corrected copy of the gene, a functional form of the protein can be produced, and affected cells, tissues, and organs may work properly. As opposed to drug-based approaches, gene therapy repairs the underlying genetic defect.

One form of gene therapy is the process of treating or alleviating diseases by genetically modifying the cells of the affected person with a new gene thats functioning properly. When a human disease gene has been recognized molecular genetics tools can be used to explore the process of the gene in both its normal and mutant states. From there, geneticists engineer a new gene that is working correctly. Then the new gene is transferred either in vivo or ex vivo and the body begins to make proteins according to the instructions in that gene. Gene therapy has to be repeated several times for the infected patient to continually be relieved, however, as repeated cell division and cell death slowly randomizes the bodys ratio of functional-to-mutant genes.

Currently, gene therapy is still being experimented with and products are not approved by the U.S. Food and Drug Administration. There have been several setbacks in the last 15 years that have restricted further developments in gene therapy. As there are unsuccessful attempts, there continue to be a growing number of successful gene therapy transfers which have furthered the research.

Major diseases that can be treated with gene therapy include viral infections, cancers, and inherited disorders, including immune system disorders.[citation needed]

Classical gene therapy is the approach which delivers genes, via a modified virus or vector to the appropriate target cells with a goal of attaining optimal expression of the new, introduced gene. Once inside the patient, the expressed genes are intended to produce a product that the patient lacks, kill diseased cells directly by producing a toxin, or activate the immune system to help the killing of diseased cells.

Nonclassical gene therapy inhibits the expression of genes related to pathogenesis, or corrects a genetic defect and restores normal gene expression.

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IPS Cell Therapy

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Regenerative Cellular Therapys natural peptide cell therapies work to optimize cell energy and nutrition. Our custom peptide compounds target the effected area and exert a preventative and curative action within the cells.

In addition to treating symptoms, our bio-medications empower the cells tissue structure and organs, blocking the connection of the symptoms to the disease and making the body fit to withstand functional stress loads. All of this is done through correcting the biological process of the diseases source and its side effects using our innovative cellular therapies. More On How Treatments Work >

Peptides are now being tested at many highly reputable universities, such as the University of California (UCLA), University of Michigan and Yale. Their research is providing more and more breakthrough uses for peptides, including treating major diseases and improving anti-aging formulas. Results for a hair re-growth peptide, and a even a peptide compound that reduces the need for insulin in diabetics, are being studied at the University of Michigan and UCLA. - Read New Articles >

These peptide breakthroughs are quickly gaining momentum, but the beneficial uses will not be realized for some time due to the lengthy and expensive FDA approval process.

Peptide treatments for many conditions, such as diabetes, cirrhosis and cancer are currently only available outside of the U.S. Regenerative Cellular Therapy provides the closest and most luxurious clinic and lodging for providing patients with these new custom Peptide treatments.

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Cell Therapy with Peptide Treatments for Cancer, Diabetes and more!

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Week 02 Cancer Genetics

By: UWS Mark Temple

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Week 02 Cancer Genetics - Video

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Aims & Scope

The importance of translatingoriginal, peer-reviewed research and review articles on the subject of cell therapy and its application to human diseases to societyhas led to the formation ofthe journalCell Medicine. To ensure high-quality contributions from all areas of transplantation, the same rigorous peer review will be applied to articles published in Cell Medicine. Articles may deal with a wide range of topics including physiological, medical, preclinical, tissue engineering, and device-oriented aspects of transplantation of nervous system, endocrine, growth factor-secreting, bone marrow, epithelial, endothelial, and genetically engineered cells, and stem cells, among others. Basic clinical studies and immunological research papers may also be featured if they have a translational interest. To provide complete coverage of this revolutionary field, Cell Medicine will report on relevant technological advances and their potential for translational medicine. Cell Medicine will be a purely online Open Access journal. There will therefore be an inexpensive publication charge, which is dependent on the number of pages, in addition to the charge for color figures. This will allow your work to be disseminated to a wider audience and also entitle you to a free PDF, as well as prepublication of an unedited version of your manuscript.

Cell Medicine features:

Original Contributions: Peer-reviewed, high-quality research investigations that represent new and significant contributions to science. Review Articles: Reviews of major areas in cellular transplantation. These may be of any length and are peer reviewed. Brief Communications: Timely and brief peer-reviewed studies. Letters to the Editor: Readers' comments on journal articles and other matters of interest to transplant researchers. Announcements and News: Notices of upcoming meetings, conferences, seminars, and other events of interest to those in the field.

Submission Requirements: From the beginning of November 2009, authors are requested to submit the original manuscript (and revised manuscript if needed) via our ManuscriptCentral websiteat http://mc.manuscriptcentral.com/cogcom-ct.

Please include a cover letter, specifying your intent to submit to Cell Medicine, as well as containing the name, address, telephone, and fax number, and electronic mail address of the author responsible for correspondence. Follow the General Form guidelines below to prepare the manuscript, figures, and tables.

At the time of submission you will be asked to confirm that you will pay the relatively inexpensive open access fees ($900 for less than 5 pages, $1800 for 5-12 pages and +$75 for each additional page) when billed. In addition, there are sections for detailing any conflicts of interest and financial support and that you (as corresponding/submitting author) have the permission of the other authors to submit the manuscript. You will be given the option of which section of the editorial office to submit to. Here you would select Cell Medicine.

There will also be a $105 submission fee.

On receipt of your manuscript, it will be checked to ensure that it is correctly formatted.

When the manuscript is accepted for publication, the author(s) will be required to provide two hard copies of the manuscript, two high-quality copies of all artwork, and a CD or disk (no zip disks) (see Final Accepted Manuscript/Disk below). Information on where to mail the final hard copy, figures, and CD/disk will be provided in an acceptance letter. Manuscripts are accepted for consideration with the understanding that they have not been published elsewhere except in abstract form and are not concurrently under review elsewhere.

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Cell Medicine - Cognizant Communication Corporation

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Our Cell Therapy Center offers advanced patented methods of stem cell treatment for different diseases and conditions. The fetal stem cells we use are nonspecialized cells able to differentiate (turn) into any other cell types forming different tissues and organs. Fetal stem cells have huge potential for differentiation and proliferation and are not rejected by the recipients body more...

Stem cell therapy has proven to be effective for organs and tissues restoration, and for fight against the incurable and obstinate diseases. We treat patients with various diseases, such as diabetes mellitus, multiple sclerosis, Parkinsons disease, Duchenne muscular dystrophy, cancer, blood diseases and many others, including rare genetic and hereditary diseases. Among our patients there are also people willing to undergo anti-aging treatment. Stem cell treatment allows for achieving effects that are far beyond the capacity of any other modern method more...

For over 19 years, we have performed more than 7,500 transplantations of fetal stem cells to people from many countries, such as the USA, China, Italy, Germany, Denmark, UAE, Egypt, Russian Federation, Greece and Cyprus, etc. Our stem cell treatments helped to prolong life and improve life quality to thousands of patients including those suffering from the incurable diseases who lost any hope for recovery.

With Cell Therapy Center EmCell located in Kiev, Ukraine, we have numerous partners in various countries devoted to provide medical advice on EmCell stem cell treatment locally.

We are always open for medical, businessandscientificcooperation.

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Stem Cell Therapy & Stem Cell Treatment - Cell Therapy Center Emcell

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SO PAULO & SAN CARLOS, Calif.--(BUSINESS WIRE)--Hospital Israelita Albert Einstein and Natera, a leading innovator in prenatal genetic testing, today announced a partnership to offer Panorama, Nateras non-invasive prenatal test. Panorama currently offers detection of chromosomal abnormalities, including trisomy 21 (Down syndrome), trisomy 13 (Patau Syndrome), trisomy 18 (Edwards syndrome), monosomy X (Turner syndrome), triploidy and vanishing twins, using only the mothers blood as early as nine weeks gestation.

Hospital Israelita Albert Einstein is committed to providing patients with the best medical care possible, and Panorama is not only a best-in-class non-invasive prenatal test but is also capable of detecting abnormalities beyond trisomies with high accuracy, said Rita Sanchez, M.D., director of maternal fetal medicine at Hospital Israelita Albert Einstein in So Paulo. This test enables us to provide expecting parents with reliable information about the genetics of their baby early on in pregnancy.

Added Matthew Rabinowitz, Ph.D., chief executive officer of Natera, Our partnership with a highly respected hospital such as Hospital Israelita Albert Einstein exemplifies Nateras commitment to delivering the highest quality of genetic tests in the world, based on accuracy, clinical coverage and fast turnaround time.

Panorama uses a simple blood draw from the mother, examines cfDNA found in maternal blood originating from both mother and fetus, and can be performed within the first trimester of pregnancy without any risk to the fetus. Panoramas technology analyzes, in a single reaction, 19,488 single nucleotide polymorphisms (SNPs), which are the most informative portions of an individuals DNA. It utilizes the NATUS [Next-generation Aneuploidy Testing Using SNPs] algorithm, an advanced version of Nateras proprietary informatics.

Across multiple clinical trials, Panorama has been validated globally for detection of trisomy 21, trisomy 18, trisomy 13, monosomy X, and now triploidy, with a sensitivity of greater than 99 percent for trisomy 21, trisomy 18, trisomy 13, and triploidy, 92 percent for monosomy X, and specificity greater than 99 percent for all syndromes tested. Panoramas clinical validation data has been reported in multiple peer-reviewed publications including the May 2013 article in Prenatal Diagnosis, authored by Professor Nicolaides, which was the first demonstration of Panoramas ability to detect triploidy. In October 2013 additional validation data was published in Fetal Diagnosis and Therapy, also authored by Professor Nicolaides, showing Panorama was able to differentiate with high accuracy between triploid and euploid cases in 56 blinded samples.

About Sociedade Beneficente Israelita Brasileira Albert Einstein (SBIBAE)

The SBIBAE operates in three integrated and equally important fronts: health care, social responsibility and the generation and dissemination of knowledge. The activities of health care are concentrated in the Hospital Israelita Albert Einstein and in the area of preventive and diagnostic medicine, which contribute to the sustainability of social responsibility, teaching and research. The Instituto Israelita Albert Einstein de Responsabilidade Social (IIRS) works in its own programs or in conjunction with public health system to help meet the health care needs, or technological skills of the community. The research and education activities are housed in the Instituto Israelita de Ensino e Pesquisa (IIEP) and confer innovation to other areas of SBIBAE.

About Natera

Natera is a leading genetic testing company that has developed a proprietary bioinformatics-based technology (NATUS) to deliver accurate and comprehensive high-throughput testing for reproductive indications from tiny quantities of DNA. Natera operates a CLIA-certified laboratory in San Carlos, Calif., providing a host of preconception and prenatal genetic testing services. Test offerings include pre-implantation genetic diagnosis to identify chromosomal anomalies or inherited genetic conditions in embryos generated during an IVF cycle; products-of-conception testing following miscarriage to rapidly and extensively analyze fetal chromosomes in order to understand the cause of the pregnancy loss; non-invasive prenatal testing to determine paternity; carrier screening tests to detect whether parents carry genetic variations that may result in disease in the child; and Panorama, a safe, simple test for pregnant women that identifies the most common chromosomal anomalies in a fetus as early as nine weeks. Natera's PreNATUS clinical trial for non-invasive screening of fetal chromosomal anomalies is funded by the NIH and is being conducted by the leaders in maternal-fetal medicine in the United States. The company was recently recognized for its work to advance prenatal care through its selection by the World Economic Forum as a 2014 Technology Pioneer. For more information, visit http://www.natera.com.

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Hospital Israelita Albert Einstein and Natera Partner to Offer Panorama™, Natera’s Non-Invasive Prenatal Test for ...

Recommendation and review posted by Bethany Smith

Oct. 30, 2013 In the first molecular genetic study of families with a history of both language impairment and autism, scientists may have uncovered a shared origin for the two conditions, an important step toward explaining why some cases of autism are accompanied by language difficulties and others are not. The study, a collaboration of The Research Institute at Nationwide Children's Hospital with experts at Rutgers University, indicates that a disorder called specific language impairment -- one of the most common developmental delays in children -- may be caused by the same genetic variants that lead to language difficulties in some children with autism. The findings are published Oct. 30 in the American Journal of Psychiatry.

As many as two-thirds of individuals with an Autism Spectrum Disorder (ASD) also have language impairments, which can range from mild limitations to complete non-verbal behavior. However, the remaining third may have normal or even above average language abilities. To investigate whether specific language impairment and language-impaired autism cases are caused by the same genetic variants, researchers examined the genetic code of 79 families with a history of both conditions.

Using a genome-wide scan and a series of language tests, the researchers identified two new genetic links for language impairment in these families: 15q23-26 and 16p12. Each of these new links is jointly related to language-impaired ASD and non-ASD related specific language impairment, suggesting a single cause for both issues.

"A genetic cause of language impairment may help explain why some kids with ASD have language impairments and others don't, as well as why some members of a family have language impairment only and others have ASD as well," says Christopher W. Bartlett, PhD, principal investigator in the Battelle Center for Mathematical Medicine at Nationwide Children's and lead author of the study. The research is part of a long-term collaboration between scientists at Nationwide Children's and Rutgers, initiated by a grant from the National Institute of Mental Health to Linda M. Brzustowicz, MD, professor of the Department of Genetics at Rutgers and senior investigator on the project.

Language impairment is not part of the diagnostic definition of ASD. And according to Dr. Bartlett, this study raises the question of whether language impairment is actually a dissociable trait in at least some forms of ASD.

"There is nothing about autism that dictates that language impairment has to occur," says Dr. Bartlett, who also is an assistant professor of pediatrics at The Ohio State University School of Medicine. "In this study, we demonstrated a shared mechanism between the two disorders. Language problems and ASD are complicated and have numerous genetic factors, but we think that many genetic factors related to communication could be the same for specific language impairment and language-impaired autism."

The genetic variations appear to be relevant to both disorders and may indicate a greater level of genetic predisposition for impairments in language ability among individuals with and without ASD in those families.

In an earlier study, the researchers found similarities in language deficit type and severity between language-impaired non-ASD and language-impaired ASD individuals in the same family. The behavioral genetics study, published in Biological Psychiatry, found that the same genes active in specific language impairment appear in ASD, but their effect is amplified in ASD. That finding, coupled with this new research, suggests that the two disorders may be on an etiological continuum.

"If further research confirms a genetic link between language impairment and ASD, then we may be able to find out why some family members only develop language impairment while others develop autism," says Dr. Bartlett. "But most of all, we want to know why there is such a range in communication abilities in children with autism -- why so many children have language difficulties when it's not required for the diagnosis."

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Scientist identify genetic link between language impairment, autism

Recommendation and review posted by Bethany Smith

In the past few years, celebrities such as Christina Applegate and Angelina Jolie have brought to the surface the subject of genetic testing for breast cancer. After learning they carried the breast cancer gene, the women decided to have radical double mastectomies.

Both women have family histories of breast and ovarian cancers. Jolies mother died in 2007 from ovarian cancer. In 1978, Applegates mother was diagnosed with breast cancer. She survived and was able to help her daughter when Christina was diagnosed 30 years later.

Many women have close family members who have been diagnosed with breast or ovarian cancer, but few have the financial and community resources available that the celebrities do.

What are the options?

Various factors can direct a patients decisions to be tested. The genetic testing Jolie and Applegate had is available in Central Illinois, but to have the tests performed is not easy. Many questions will need to be answered by healthcare providers and the patient.

Why are you testing?

First, the patient will have to assess the need for the test.

What is really leading you to have testing? Daniel Groeppe, genetic counselor at the SIU School of Medicine may ask. Is there something about your family that youre concerned about or did you hear about it in the media? If it is the latter, then our conversation is mostly going to be about what are your chances you could even develop breast cancer based on a normal or a negative family history.

The first step is to develop a cancer family tree. All family members and all cancers should be recorded. The BRCA gene can be inherited through the father, warns SIU School of Medicine Director of Community Support, Cindy Davidsmeyer.

The BRCA1 and BRCA2 are the two genes related to breast cancer. People start off with a basic set of genes inherited from their parents. I like to think of them as instructions; kind of like the blue print, Groeppe said. If there are any changes in those instructions, we call it a mutation. When you have these mutations, then you have an increased risk for breast cancer and ovarian cancer.

Read this article:
Do homework before seeking genetic tests

Recommendation and review posted by Bethany Smith

PUBLIC RELEASE DATE:

31-Oct-2013

Contact: Judy Romero jromero@crg.org Cardiovascular Research Foundation

SAN FRANCISCO, CA October 31, 2013 According to a new study, genetic profiling of patients undergoing percutaneous coronary intervention (PCI) may help cardiology teams adjust treatment and improve ischemic outcomes for patients that do not properly metabolize thienopyridine blood thinning therapies such as clopidogrel.

Findings from the GIANT trial were presented today at the 25th annual Transcatheter Cardiovascular Therapeutics (TCT) scientific symposium. Sponsored by the Cardiovascular Research Foundation (CRF), TCT is the world's premier educational meeting specializing in interventional cardiovascular medicine.

The effectiveness of clopidogrel depends on activation to an active metabolite, principally via the CYP2C19 enzymatic pathway. Acute coronary syndrome patients that carry a CYP2C19 gene variant poorly metabolize the drug. These patients are known as slow responders and exhibit a higher one year risk of major ischemic events following PCI. Genetic tests can help identify a patient's CYP2C19 genotype, but it is unknown if on-line adjustment of thienopyridine therapy in the genetically slow-responder patient population may counteract this outcome.

The GIANT trial evaluated the clinical impact of CYP2C19 genetic profiling and compliance to an adjusted thienopyridine treatment. The primary endpoint was a composite of death, myocardial infarction, and stent thrombosis after one year in slow responder patients with appropriate therapy after genotyping, compared to non-slow responders.

The prospective, multicenter, single arm study enrolled 1,499 patients at the time of primary PCI (onset chest pain < 24 hours). Genetic profiling was performed within 48 hours after intervention to detect a loss of CYP2C19 gene function and identify a resistance to clopidogrel.

Strong recommendations for treatment adjustment were sent to investigators when patients were identified as slow responders.

Dual antiplatelet therapy (DAPT) was prescribed for 12 months after PCI and one year follow up was performed in 96.4 percent of patients (n=1,445) including objective assessment of compliance. A total of 22 percent of patients (n=319) had a profile associated with a CYP2C19 loss of function, known as the slow responder group. The remaining patients constituted the control group.

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Results of the GIANT trial reported at TCT 2013

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Lets talk genetics

By: sambo20091

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Lets talk genetics - Video

Recommendation and review posted by Bethany Smith

LOS ANGELES, Oct. 31, 2013 (GLOBE NEWSWIRE) -- Response Genetics, Inc. (RGDX), a company focused on the development and sale of molecular diagnostic tests that help determine a patient's response to cancer therapy, will announce its third quarter 2013 financial results and give an operational update in a press release to be issued before the market opens on Thursday, November 7, 2013. The company will host a conference call that same day at 10:00 a.m. EST to discuss its financial results.

CONFERENCE CALL DETAILS

To access the conference call by phone on November 7 at 10:00 a.m. EST, dial (800) 537-0745 or (253) 237-1142 for international participants. A telephone replay will be available beginning approximately two hours after the call through November 9, 2013, and may be accessed by dialing (855) 859-2056 or (404) 537-3406. The conference passcode for both the live call and replay is 94263838.

To access the live and archived webcast of the conference call, go to the Investor Relations section of the Company's Web site at http://investor.responsegenetics.com/events.cfm. It is advised that participants connect at least 15 minutes prior to the call to allow for any software downloads that might be necessary.

About Response Genetics, Inc.

Response Genetics, Inc. (the "Company") is a CLIA-certified clinical laboratory focused on the development and sale of molecular diagnostic testing services for cancer. The Company's technologies enable extraction and analysis of genetic information derived from tumor cells stored as formalin-fixed and paraffin-embedded specimens. The Company's principal customers include oncologists and pathologists. In addition to diagnostic testing services, the Company generates revenue from the sale of its proprietary analytical pharmacogenomic testing services of clinical trial specimens to the pharmaceutical industry. The Company's headquarters is located in Los Angeles, California. For more information, please visit http://www.responsegenetics.com.

Forward-Looking Statement Notice

Except for the historical information contained herein, this press release and the statements of representatives of the Company related thereto contain or may contain, among other things, certain forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995.

Such forward-looking statements involve significant risks and uncertainties. Such statements may include, without limitation, statements with respect to the Company's plans, objectives, projections, expectations and intentions, such as the ability of the Company, to provide clinical testing services to the medical community, to continue to strengthen and expand its sales force, to continue to build its digital pathology initiative, to attract and retain qualified management, to continue to strengthen marketing capabilities, to expand the suite of ResponseDX(R) products, to continue to provide clinical trial support to pharmaceutical clients, to enter into new collaborations with pharmaceutical clients, to enter into areas of companion diagnostics, to continue to execute on its business strategy and operations, to continue to analyze cancer samples and the potential for using the results of this research to develop diagnostic tests for cancer, the usefulness of genetic information to tailor treatment to patients, and other statements identified by words such as "project," "may," "could," "would," "should," "believe," "expect," "anticipate," "estimate," "intend," "plan" or similar expressions.

These statements are based upon the current beliefs and expectations of the Company's management and are subject to significant risks and uncertainties, including those detailed in the Company's filings with the Securities and Exchange Commission. Actual results, including, without limitation, actual sales results, if any, or the application of funds, may differ from those set forth in the forward-looking statements. These forward-looking statements involve certain risks and uncertainties that are subject to change based on various factors (many of which are beyond the Company's control). The Company undertakes no obligation to publicly update forward-looking statements, whether because of new information, future events or otherwise, except as required by law.

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Response Genetics, Inc. to Release Third Quarter 2013 Financial Results and Host Conference Call on November 7, 2013

Recommendation and review posted by Bethany Smith

STAMFORD, Conn. -- Stamford's Alliance for Cancer Gene Therapy's "Achieving Cancer Remission with Cell and Gene Therapies" event attracted more than 100 people to New York City last week.

More than 100 donors, scientists, biotech representatives and physicians attended the Tuesday night event at the Harvard Club of New York City, according to a news release.The event "highlighted recent tremendous strides made in combating cancer with cell and gene therapy treatments, and served as appreciation for donors who have committed time and funds to furthering research and clinical trials across the nation," according to the release.

Our donors have allowed top scientific minds to explore this new and promising avenue of cancer treatment, and their philanthropy is directly linked to the lives saved so far, said Barbara Netter, who co-founded the alliance in 2001 with her husband, Edward, in the release.

Netter later said that "much additional research needs to be funded in order to achieve the goal of the fully successful treatment of all types of cancer," according to the release. Netter has assumed the mantle of president of ACGT to "chart a strategic course for the organizations continued success" and further the goal, according to the release.

Guests at the evening event were treated to a reception at the Harvard Club, followed by a salutation from host Dr. Savio Woo, according to the release.

"Dr. Woo Chairman of ACGTs Scientific Advisory Council and Professor of Hematology and Oncology at the Tisch Cancer Institute at Mount Sinai School of Medicine in New York City was instrumental in ACGTs founding over a decade ago," representatives said in the release.

Connie Burnett-West, a cancer survivor "who overcame a critical case lung cancer with gene and cell therapy treatment," also attended the event, according to the release.

Surgery and radiation werent options, and I was told I had limited hope for recovery, Burnett-West said in the release. But after a sixth-month course of gene therapy, Ive been in remission for over 10 years. I could not have imagined a treatment so easy and effective.

The evening also featured a presentation from three of ACGTs Research Fellows, including Carl H. June (M.D., University of Pennsylvania), Laurence Cooper (M.D., Ph.D., MD Anderson Cancer Center) and Michel Sadelain (M.D., Ph.D., Memorial Sloan-Kettering Cancer Center), according to the release. The three "spoke of the breakthroughs and growing momentum that gene and cell therapy has achieved with the support of ACGT," according to the release.

ACGT has the potential to provide less expensive and less harrowing cancer treatment and, ultimately, a cure, Dr. Carl June said in the release. And all of ACGTs life-saving work was funded through philanthropy.

More here:
Stamford's Alliance For Cancer Gene Therapy Celebrates In NYC

Recommendation and review posted by Bethany Smith

Multiple Sclerosis (MS) is the most common disabling neurological disease of young adults. It most often appears when people are between 20 to 40 years old. However, it can also affect children and older people.

The course of MS is unpredictable. A small number of those with MS will have a mild course with little to no disability, while another smaller group will have a steadily worsening disease that leads to increased disability over time. Most people with MS, however, will have short periods of symptoms followed by long stretches of relative relief, with partial or full recovery. There is no way to predict, at the beginning, how an individual persons disease will progress.

Researchers have spent decades trying to understand why some people get MS and others don't, and why some individuals with MS have symptoms that progress rapidly while others do not. How does the disease begin? Why is the course of MS so different from person to person? Is there anything we can do to prevent it? Can it be cured?

This brochure includes information about why MS develops, how it progresses, and what new therapies are being used to treat its symptoms and slow its progression. New treatments can reduce long-term disability for many people with MS. However, there are still no cures and no clear ways to prevent MS from developing.

Multiple sclerosis (MS) is a neuroinflammatory disease that affects myelin , a substance that makes up the membrane (called the myelin sheath) that wraps around nerve fibers (axons). Myelinated axons are commonly called white matter. Researchers have learned that MS also damages the nerve cell bodies, which are found in the brains gray matter, as well as the axons themselves in the brain, spinal cord, and optic nerve (the nerve that transmits visual information from the eye to the brain). As the disease progresses, the brains cortex shrinks (cortical atrophy).

The term multiple sclerosis refers to the distinctive areas of scar tissue (sclerosis or plaques) that are visible in the white matter of people who have MS. Plaques can be as small as a pinhead or as large as the size of a golf ball. Doctors can see these areas by examining the brain and spinal cord using a type of brain scan called magnetic resonance imaging (MRI).

While MS sometimes causes severe disability, it is only rarely fatal and most people with MS have a normal life expectancy.

Plaques, or lesions, are the result of an inflammatory process in the brain that causes immune system cells to attack myelin. The myelin sheath helps to speed nerve impulses traveling within the nervous system. Axons are also damaged in MS, although not as extensively, or as early in the disease, as myelin.

Under normal circumstances, cells of the immune system travel in and out of the brain patrolling for infectious agents (viruses, for example) or unhealthy cells. This is called the "surveillance" function of the immune system.

Surveillance cells usually won't spring into action unless they recognize an infectious agent or unhealthy cells. When they do, they produce substances to stop the infectious agent. If they encounter unhealthy cells, they either kill them directly or clean out the dying area and produce substances that promote healing and repair among the cells that are left.

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Multiple Sclerosis: Hope Through Research: National Institute of ...

Recommendation and review posted by simmons

The Gene Therapy Research Program is focused on the development of gene therapy and genetic engineering technologies for the application to cancer, transplantation, and regenerative medicine.

The main focus is cancer gene therapy. It is a promising new approach in which genes delivered directly to cancer cells will serve as the blueprint for therapeutic proteins that kill the cells from within or will provoke an immune response so that the body rejects the cells. While this method promises to be more effective with fewer side effects than current chemotherapy, the efficiency of delivering genes has proven to be a major obstacle to its success. Many strategies for gene therapy have involved the use of certain viruses as vehicles ("vectors") for gene delivery, because viruses have evolved efficient ways to insert their genes into human cells. By removing the viral genes that naturally allowed the virus to spread from cell to cell, these vectors were made safer; however, over the last decade, it has been found that the efficiency of these disabled viruses in infecting tumors and delivering genes is too low to be therapeutically useful.

Dr. Noriyuki Kasahara was one of the first to demonstrate that by making retrovirus vectors that are less disabled and hence can take advantage of the natural process of virus replication and spread from cell to cell, their efficiency is greatly improved for gene delivery targeted to human cancers. His group is now leading a consortium that is funded by a multi-institutional U01 award from the NIH; it includes collaborating groups at UC San Francisco, the University of Southern California, the National Gene Vector Biorepository, and biotech partner Tocagen Inc. in the development of this tumor-selective replication-competent retrovirus (RCR) vector system for clinical use. The Investigational New Drug (IND) application to conduct a first-in-man Phase I clinical trial for the RCR vector-mediated suicide gene therapy in brain tumor patients wasapproved by the Food and Drug Administration (FDA), and this trial was initiated at UCLA in August 2010. Read Fall 2012 update on "Dr. Kasahara's group is now leading a consortium that has initiated a first-in-human clinical trial to test this novel gene therapy technology in brain cancer patients"

Other projects ongoing in the Gene Therapy Research Program focus on emulating and adapting the mechanisms used by tumors to evade or suppress the immune system and applying them toward achieving long-term graft survival in cellular transplantation. Dr. Kasahara's group was among the first to demonstrate that this capability can be achieved by using the gene transfer of small interfering RNA (siRNA) to silence specific tissue antigens (HLA) and thereby enhance the histocompatibility of donor cells to be transplanted into HLA-mismatched recipients. Furthermore, it is increasingly apparent that human adult and embryonic stem-cell-derived tissues, which do not initially express HLA, subsequently increase their levels of HLA expression as they differentiate into mature tissues. Hence, this strategy also has significant implications for regenerative medicine: Any non-autologous adult stem-cell-derived tissues and every embryonic stem-cell-derived tissue will eventually encounter the same problems of immune rejection that have long confronted the field of adult organ transplantation, and it is hoped that this strategy may represent an effective and fundamentally different approach to overcoming such problems by genetically modifying the donor-derived graft cells to evade the recipient's immune system instead of immunosuppressing the recipient host.

Learn more about our team

Noriyuki Kasahara, MD, PhDDirector, JCCC Vector Shared Resource & CURE Vector Core Facility Professor, Departments of Medicine and Molecular & Medical Pharmacology

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UCLA | Gene Research Therapy, Noriyuki Kasahara MD PhD ...

Recommendation and review posted by Bethany Smith

Center for Cell and Gene Therapy, Baylor College of Medicine, Houston

Collaboration between Baylor College of Medicine, The Methodist Hospital and Texas Children's Hospital. Clinical research in the areas of stem cell transplantation, cellular therapy, and gene therapy.

The Center fosters a multidisciplinary approach to new research as well as collaborative research endeavors in the area of gene therapy. The Vector Core manufactures several recombinant viral vectors.

The research is focused on various aspects of gene therapy, such as understanding basic virology, efficient gene delivery into the nucleus of cells, and incorporation of these genes into the genome.

Dr. Curiel heads the center and is engaged in advancing the application of gene transfer methods for treatment of human disease. The efforts are focused on the development of improved vectors to accomplish targeted, cell-specific delivery.

Research in the laboratory has centered on the molecular biology of adeno-associated virus (AAV) in order to exploit the unique features of this virus to develop an efficient viral vector system for use in human gene therapy.

The Harvard Gene Therapy Initiative is headed by Dr. Richard Mulligan with the objective of promoting the use of gene therapy and to conduct research developing new gene delivery vector technologies.

Diseases of the lung, cardiovascular system, muscles, brain, and skin are focus areas of research as well as the development of gene therapy vectors and the identification of disease-causing genes.

The major goals of the Innovative Cancer Therapies Program will be to enhance communication and collaboration among investigators and to develop new funding opportunities.

The program has brought together regulatory, quality, product development, manufacturing and facilities engineering expertise to enable the translation of novel, experimental research into medicine for use in human clinical trials.

Original post:
Gene Therapy Research Institutes and Universities

Recommendation and review posted by Bethany Smith

Researchers are looking at different ways of using gene therapy, including

Some types of gene therapy aim to boost the body's natural ability to attack cancer cells. Our immune system has cells that recognise and kill harmful things that can cause disease, such as cancer cells.

There are many different types of immune cell. Some of them produce proteins that encourage other immune cells to destroy cancer cells. Some types of therapy add genes to a patient's immune cells to make them better at finding or destroying particular types of cancer. There are a few trials using this type of gene therapy in the UK.

Some gene therapies put genes into cancer cells to make the cells more sensitive to particular treatments such as chemotherapy or radiotherapy. This type of gene therapy aims to make the other cancer treatments work better.

Some types of gene therapy deliver genes into the cancer cells that allow the cells to change drugs from an inactive form to an active form. The inactive form of the drug is called a pro drug.

After giving the carrier containing the gene, the doctor gives the patient the pro drug. The pro drug may be a tablet or capsule that you swallow, or you may have it into the bloodstream.

The pro drug circulates in the body and doesn't harm normal cells. But when it reaches the cancer cells, the gene activates it and the drug kills the cancer cells.

Some gene therapies block processes that cancer cells use to survive. For example, most cells in the body are programmed to die if their DNA is damaged beyond repair. This is called programmed cell death or apoptosis. But cancer cells block this process so they don't die even when they are supposed to. Some gene therapy strategies aim to reverse this blockage. Doctors hope that these new types of treatment will make the cancer cells die.

Some viruses infect and kill cells. Researchers are working on ways to change these viruses so that they only target and kill cancer cells, leaving healthy cells alone. This sort of treatment uses the viruses to kill cancer cells directly rather than to deliver genes. So it is not cancer gene therapy in the true sense of the word. But doctors sometimes refer to it as gene therapy.

One example of this type of research uses the cold sore virus (herpes simplex virus). The changed virus is called Oncovex. It has been tested in early clinical trials for advanced melanoma, pancreatic cancer and head and neck cancers.

More:
Gene therapy : Cancer Research UK : CancerHelp UK

Recommendation and review posted by Bethany Smith

Oct. 30, 2013 Glioblastoma multiforme (GBM), the brain cancer that killed Sen. Edward Kennedy and kills approximately 13,000 Americans a year, is aggressive and incurable. Now a Northwestern University research team is the first to demonstrate delivery of a drug that turns off a critical gene in this complex cancer, increasing survival rates significantly in animals with the deadly disease.

The novel therapeutic, which is based on nanotechnology, is small and nimble enough to cross the blood-brain barrier and get to where it is needed -- the brain tumor. Designed to target a specific cancer-causing gene in cells, the drug simply flips the switch of the troublesome oncogene to "off," silencing the gene. This knocks out the proteins that keep cancer cells immortal.

In a study of mice, the nontoxic drug was delivered by intravenous injection. In animals with GBM, the survival rate increased nearly 20 percent, and tumor size was reduced three to four fold, as compared to the control group. The results will be published Oct. 30 in Science Translational Medicine.

"This is a beautiful marriage of a new technology with the genes of a terrible disease," said Chad A. Mirkin, a nanomedicine expert and a senior co-author of the study. "Using highly adaptable spherical nucleic acids, we specifically targeted a gene associated with GBM and turned it off in vivo. This proof-of-concept further establishes a broad platform for treating a wide range of diseases, from lung and colon cancers to rheumatoid arthritis and psoriasis."

Mirkin is the George B. Rathmann Professor of Chemistry in the Weinberg College of Arts and Sciences and professor of medicine, chemical and biological engineering, biomedical engineering and materials science and engineering.

Glioblastoma expert Alexander H. Stegh came to Northwestern University in 2009, attracted by the University's reputation for interdisciplinary research, and within weeks was paired up with Mirkin to tackle the difficult problem of developing better treatments for glioblastoma.

Help is critical for patients with GBM: The median survival rate is 14 to 16 months, and approximately 16,000 new cases are reported in the U.S. every year.

In their research partnership, Mirkin had the perfect tool to tackle the deadly cancer: spherical nucleic acids (SNAs), new globular forms of DNA and RNA, which he had invented at Northwestern in 1996, and which are nontoxic to humans. The nucleic acid sequence is designed to match the target gene.

And Stegh had the gene: In 2007, he and colleagues identified the gene Bcl2Like12 as one that is overexpressed in glioblastoma tumors and related to glioblastoma's resistance to conventional therapies.

"My research group is working to uncover the secrets of cancer and, more importantly, how to stop it," said Stegh, a senior co-author of the study. "Glioblastoma is a very challenging cancer, and most chemo-therapeutic drugs fail in the clinic. The beauty of the gene we silenced in this study is that it plays many different roles in therapy resistance. Taking the gene out of the picture should allow conventional therapies to be more effective."

Read more here:
Incurable brain cancer gene silenced: Gene regulation technology increases survival rates in mice with glioblastoma

Recommendation and review posted by Bethany Smith

By Steven Reinberg HealthDay Reporter

WEDNESDAY, Oct. 30 (HealthDay News) -- Chances of surviving lung cancer longer increase when treatment is personalized based on the genetics of the cancer, German researchers report.

Knowing the tumor's genetic signature can help doctors spot differences in cancer cells that may lead to a more accurate diagnosis and better-targeted therapy, the researchers explained.

"Gene classification and diagnosis has a profound impact on patients' survival," said study co-author Dr. Reinhard Buttner, a professor of pathology at University Hospital Cologne.

"Our data were collected from approximately 5,100 lung cancer patients and show that genotyping of lung cancer doubles overall survival in patients with two specific mutations -- EGFR-mutated and ALK-translocated," Buttner said.

Patients with those mutations who received personalized therapies showed survival advantages ranging somewhere between 12 months and 21 months.

The report was published Oct. 30 in the journal Science Translational Medicine.

The researchers found that although some lung cancer cells look the same under the microscope, they may actually be quite different genetically.

"Systematic profiling of gene mutations in lung cancer allows precise classification and diagnostics and predicts the efficacy of targeted and personalized therapies. Every lung cancer should be analyzed for mutations to find the best therapy," Buttner said.

Surprisingly, looking at two common types of lung cancer -- small-cell and large-cell -- from a genetic perspective actually ended up eliminating large-cell lung cancer as a distinct category, he said.

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Gene Testing May Boost Lung Cancer Survival: Study

Recommendation and review posted by Bethany Smith

Oct. 31, 2013 Researchers at Johns Hopkins say they have found that a gene already implicated in human speech disorders and epilepsy is also needed for vocalizations and synapse formation in mice. The finding, they say, adds to scientific understanding of how language develops, as well as the way synapses the connections among brain cells that enable us to think are formed. A description of their experiments appears in Science Express on Oct. 31.

A group led by Richard Huganir, Ph.D., director of the Solomon H. Snyder Department of Neuroscience and a Howard Hughes Medical Institute investigator, set out to investigate genes involved in synapse formation. Gek-Ming Sia, Ph.D., a research associate in Huganirs laboratory, first screened hundreds of human genes for their effects on lab-grown mouse brain cells. When one gene, SRPX2, was turned up higher than normal, it caused the brain cells to erupt with new synapses, Sia found.

When Huganirs team injected fetal mice with an SRPX2-blocking compound, the mice showed fewer synapses than normal mice even as adults, the researchers found. In addition, when SRPX2-deficient mouse pups were separated from their mothers, they did not emit high-pitched distress calls as other pups do, indicating they lacked the rodent equivalent of early language ability.

Other researchers analyses of the human genome have found that mutations in SRPX2 are associated with language disorders and epilepsy, and when Huganirs team injected the human SRPX2 with the same mutations into the fetal mice, they also had deficits in their vocalization as young pups.

Another research group at Institut de Neurobiologie de la Mditerrane in France had previously shown that SRPX2 interacts with FoxP2, a gene that has gained wide attention for its apparently crucial role in language ability. Huganirs team confirmed this, showing that FoxP2 controls how much protein the SRPX2 gene makes and may affect language in this way. FoxP2 is famous for its role in language, but its actually involved in other functions as well, Huganir comments. SRPX2 appears to be more specialized to language ability. Huganir suspects that the gene may also be involved in autism, since autistic patients often have language impairments, and the condition has been linked to defects in synapse formation.

This study is only the beginning of teasing out how SRPX2 acts on the brain, Sia says. Wed like to find out what other proteins it acts on, and how exactly it regulates synapses and enables language development.

Roger Clem of the Mount Sinai School of Medicine also participated in the study.

This study was supported by the National Institute of Mental Health (grant number P50MH084020) and the National Institute of Neurological Disorders and Stroke (grant number NS050274).

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Gene found to foster synapse formation in the brain

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PUBLIC RELEASE DATE:

30-Oct-2013

Contact: Megan Fellman fellman@northwestern.edu 847-491-3115 Northwestern University

Glioblastoma multiforme (GBM), the brain cancer that killed Sen. Edward Kennedy and kills approximately 13,000 Americans a year, is aggressive and incurable. Now a Northwestern University research team is the first to demonstrate delivery of a drug that turns off a critical gene in this complex cancer, increasing survival rates significantly in animals with the deadly disease.

The novel therapeutic, which is based on nanotechnology, is small and nimble enough to cross the blood-brain barrier and get to where it is needed -- the brain tumor. Designed to target a specific cancer-causing gene in cells, the drug simply flips the switch of the troublesome oncogene to "off," silencing the gene. This knocks out the proteins that keep cancer cells immortal.

In a study of mice, the nontoxic drug was delivered by intravenous injection. In animals with GBM, the survival rate increased nearly 20 percent, and tumor size was reduced three to four fold, as compared to the control group. The results will be published Oct. 30 in Science Translational Medicine.

"This is a beautiful marriage of a new technology with the genes of a terrible disease," said Chad A. Mirkin, a nanomedicine expert and a senior co-author of the study. "Using highly adaptable spherical nucleic acids, we specifically targeted a gene associated with GBM and turned it off in vivo. This proof-of-concept further establishes a broad platform for treating a wide range of diseases, from lung and colon cancers to rheumatoid arthritis and psoriasis."

Mirkin is the George B. Rathmann Professor of Chemistry in the Weinberg College of Arts and Sciences and professor of medicine, chemical and biological engineering, biomedical engineering and materials science and engineering.

Glioblastoma expert Alexander H. Stegh came to Northwestern University in 2009, attracted by the University's reputation for interdisciplinary research, and within weeks was paired up with Mirkin to tackle the difficult problem of developing better treatments for glioblastoma.

Help is critical for patients with GBM: The median survival rate is 14 to 16 months, and approximately 16,000 new cases are reported in the U.S. every year.

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Incurable brain cancer gene is silenced

Recommendation and review posted by Bethany Smith