SCIENCE NEWS Mental Illness Tied to Immune Defect — Bone Marrow Transplants Cure Mice of Hair-Pulling Compulsion A geneticist discovered that bone marrow transplants cure mutant mice who pull out their hair compulsively. The study provides the first cause-and-effect link between immune system cells and mental illness, and points toward eventual new psychiatric treatments. [Press release from the University of Utah discussing online prepublication in Cell]Tekmira Reports Complete Protection from Deadly Ebola Virus in Nonhuman Primates with SNALP-RNAi Tekmira Pharmaceuticals Corporation announced the publication of a series of studies demonstrating the ability of an RNAi therapeutic utilizing Tekmira’s lipid nanoparticle technology, SNALP, to protect nonhuman primates from Ebola virus. [Press release from Tekmira Pharmaceuticals Corporation discussing online prepublication in Lancet] Novel RNA Interference Screening Technique Identifies Possible Path for Malignant Glioma Treatment Researchers report a cellular pathway in the deadly brain cancer malignant glioma, a pathway essential to the cancer’s ability to grow – and a potential target for therapy that would stop the cancer’s ability to thrive. [Press release from the University of Massachusetts Medical School discussing online prepublication in Nature Medicine] Gene Fusions May Be the ‘Smoking Gun’ in Prostate Cancer Development The study suggests that the gene fusion – not the androgen receptor – is a more specific “bad actor” in prostate cancer and is the real smoking gun that should be targeted by treatments. [Press release from the University of Michigan Comprehensive Cancer Center discussing online prepublication in Cancer Cell] Transplant Tolerance 'Signature' Identified Researchers have identified a ‘full set’ of immunological markers in the blood, which could be used to predict whether an individual’s kidney transplant will be a long term success or whether it will fail. [Press release from King’s College London discussing online prepublication in the Journal of Clinical Investigation] Gene Pattern May Identify Kidney Transplant Recipients Who Don’t Need Life-Long Anti-Rejection Drugs Researchers have identified a distinct pattern of gene expression in the largest reported group of kidney transplant recipients who have not rejected the transplant kidneys even though they stopped taking anti-rejection drugs. [Press release from the National Institutes of Health discussing online prepublication in the Journal of Clinical Investigation] Novel Stem Cells Could Offer Myocardial Infarct Therapy Stem cells derived from the human amniotic sac can become beating heart muscle cells, at least in rats, researchers said, offering the possibility of off-the-shelf stem cell therapy for heart attacks. [Press release from MedPage Today discussing online prepublication in Circulation Research] To Attack H1N1, Other Flu Viruses, Gold Nanorods Deliver Potent Payload Future pandemics of seasonal flu, H1N1 and other drug-resistant viruses may be thwarted by a potent, immune-boosting payload that is effectively delivered to cells by gold nanorods. [Press release from the University at Buffalo discussing online prepublication in the Proceedings of the National Academy of Sciences USA] Rare Hybrid Cell Key to Regulating the Immune System A cell small in number but powerful in its ability to switch the immune system on or off is a unique hybrid of two well-known immune cell types, researchers report. [Press release from the University of Georgia discussing online prepublication in the Proceedings of the National Academy of Sciences USA] Novel Therapeutic Approach Shows Promise Against Multiple Bacterial Pathogens Researchers combined components isolated from the membrane of a weakened strain of Francisella tularensis, the agent of tularemia, with the Juvaris product CLDC (cationic liposome DNA complexes). The combination stimulated a natural antibacterial mechanism, reactive oxygen species (ROS) and reactive nitrogen species, in immune cells that ingest bacteria. [Press release from the National Institute of Allergy and Infectious Diseases discussing online prepublication in PLoS Pathogens] Retina Created from Human Stem Cells Scientists have created an eight-layer, early-stage retina from human embryonic stem cells, the first three-dimensional tissue structure to be made from stem cells. [Press release from the University of California, Irvine discussing online prepublication in the Journal of Neuroscience Methods] Suicide Genes May Improve Safety of Stem Cell Therapies Cells programmed to commit immediate suicide may increase the safety of cell therapy treatments for patients with cancer and other diseases. [American Society of Gene & Cell Therapy Press Release] Gene Therapy Reverses Muscle Weakness in Muscular Dystrophy Patients In a small study, patients with limb-girdle muscular dystrophy received a transplant of healthy genes. After six months, a majority of patients showed increased levels of muscle fiber and gene expression similar to those in healthy patients with normal muscle control. [American Society of Gene & Cell Therapy Press Release] Chemotherapy Ups Odds for Success in Treating “Bubble Boy” Disease Gene therapy combined with chemotherapy may lead to immune system improvement in patients with bubble boy disease. [American Society of Gene & Cell Therapy Press Release] DNA Vaccine Eases Treatment of Hepatitis C A vaccine that delivers corrected DNA to the immune system may hold the cure for hepatitis C, according to results of a first-of-its-kind study. [American Society of Gene & Cell Therapy Press Release] IV Is Key for Treatment of Advanced Cancers Intravenous (IV) delivery of a cancer-targeting virus is successful in destroying cancer cells that have spread throughout the body. [American Society of Gene & Cell Therapy Press Release] Amsterdam Molecular Therapeutics Reports Promising Data from Cholesterol Lowering Gene Therapy Study Amsterdam Molecular Therapeutics reported positive preclinical data from a study using an AAV gene therapy product to lower cholesterol. [Press release from Amsterdam Molecular Therapeutics discussing research presented at the 13th Annual American Society of Gene & Cell Therapy Meeting] Targeted Immunotherapy Shows Promise in Penn Medicine Studies for Metastatic Breast and Pancreatic Cancers Early trials using targeted monoclonal antibodies in combination with existing therapies show promise in treating pancreatic cancer and metastatic breast cancer. [Press release from the University of Pennsylvania School of Medicine discussing research to be presented at the 2010 American Society of Clinical Oncology Annual Meeting] Gene Therapy May Be Effective in Treating PAH Gene therapy has been shown to have positive effects in rat models of pulmonary arterial hypertension (PAH), according to researchers at the University of Adelaide in Australia. [Press release from EurekAlert! discussing research presented at the American Thoracic Society 2010 International Conference] Mouse Project to Find Each Gene's Role An ambitious effort by the International Mouse Phenotyping Consortium to identify the function of every gene in the mouse genome looks set to provide scientists with the ultimate mouse model of human disease. [Nature News] Researchers Successfully Culture Liver Stem Cells Outside the Body for the First Time A research team at the National Cancer Center is the world's first to successfully stabilize and culture liver stem cells outside the body. [Press release from The Mainichi Daily News] Phase II Clinical Trial Data Show Significant Improvements in Outcomes and Symptoms in Advanced Heart Failure Patients Treated with Celladon’s Genetically Targeted Enzyme Replacement Therapy MYDICAR® Celladon Corp. announced that six-month data from its Phase II clinical trial of MYDICAR® show improvements in clinical outcomes and disease markers in advanced heart failure patients treated with the genetically targeted enzyme replacement therapy. [Celladon Corporation Press Release] CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal) CD95 Promotes Tumor Growth The results demonstrate that CD95 has a growth-promoting role during tumorigenesis and indicate that efforts to inhibit its activity rather than to enhance it should be considered during cancer therapy. [Nature] Hematopoietic Origin of Pathological Grooming in Hoxb8 Mutant Mice It has been suggested that the grooming dysfunction results from a nociceptive defect, also exhibited by Hoxb8 mutant mice. However, bone marrow transplant experiments and cell type-specific disruption of Hoxb8 reveal that these two phenotypes are separable, with the grooming phenotype derived from the hematopoietic lineage and the sensory defect derived from the spinal cord cells. [Cell] A Genome-Wide RNA Interference Screen Reveals an Essential CREB3L2-ATF5-MCL1 Survival Pathway in Malignant Glioma with Therapeutic Implications The results reveal an essential survival pathway in malignant glioma, whereby activation of a RAS–mitogen-activated protein kinase or phosphoinositide-3-kinase signaling cascade leads to induction of the transcription factor cAMP response element–binding protein-3–like-2 (CREB3L2), which directly activates ATF5 expression. ATF5, in turn, promotes survival by stimulating transcription of myeloid cell leukemia sequence-1 (MCL1), an antiapoptotic B cell leukemia-2 family member. [Nat Med] An Autoimmune-Mediated Strategy for Prophylactic Breast Cancer Vaccination Researchers found that immunoreactivity against alpha-lactalbumin provides substantial protection and therapy against growth of autochthonous tumors in transgenic mouse models of breast cancer and against 4T1 transplantable breast tumors in BALB/c mice. [Nat Med] An Integrated Network of Androgen Receptor, Polycomb, and TMPRSS2-ERG Gene Fusions in Prostate Cancer Progression The findings provide a working model in which TMPRSS2-ERG plays a critical role in cancer progression by disrupting lineage-specific differentiation of the prostate and potentiating the EZH2-mediated dedifferentiation program. [Cancer Cell] Identification of a B Cell Signature Associated with Renal Transplant Tolerance in Humans The results point to a critical role for B cells in regulating alloimmunity and provide a candidate set of genes for wider-scale screening of renal transplant recipients. [J Clin Invest] Xenografted Human Amniotic Membrane–Derived Mesenchymal Stem Cells Are Immunologically Tolerated and Transdifferentiated into Cardiomyocytes Because human amniotic membrane–derived mesenchymal cells have a high ability to transdifferentiate into cardiomyocytes and to acquire immunologic tolerance in vivo, they can be a promising cellular source for allograftable stem cells for cardiac regenerative medicine. [Circ Res] Gold Nanorod Delivery of an ssRNA Immune Activator Inhibits Pandemic H1N1 Influenza Viral Replication Researchers introduce a nanotechnology approach for the therapy of pandemic and seasonal influenza virus infections, which uses gold nanorods to deliver an innate immune activator, producing a localized therapeutic response. [Proc Natl Acad Sci U S A] B-Lymphoid Cells with Attributes of Dendritic Cells Regulate T Cells via Indoleamine 2,3-Dioxygenase Researchers show that indoleamine 2,3-dioxygenase-competent cells express the B-lineage commitment factor Pax5 and surface immunoglobulins. [Proc Natl Acad Sci U S A] Effective, Broad Spectrum Control of Virulent Bacterial Infections Using Cationic DNA Liposome Complexes Combined with Bacterial Antigens Development of novel, immunomodulatory prophylaxes that target the host immune system, rather than the invading microbe, could serve as effective alternatives to traditional chemotherapies. Researchers describe the development and mechanism of a novel pan-anti-bacterial prophylaxis. [PLoS Pathog]
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