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Application Stories

Previous recipients of the Exiqon Grant Program

We have had the honor of supporting many excellent projects with our Grant Program. Below is a presentation of previous years' grant recipients.


miRNAs derived from urinary exosomes as non-invasive biomarkers for diabetic nephropathy

Dr. Rajeev Rohatgi is Assistant Professor of Medicine and Pediatrics at The Mount Sinai School of Medicine, New York. His research focuses on the identification of biomarkers for the progression of diabetic nephropathy (DN), a condition which affects about 30% of patients with type 2 diabetes. His lab has developed a novel way of recovering exosomes from urine. The proposed project will aim to identify exosomal microRNA biomarkers in urine derived from patients with type 2 diabetes and correlate these with the severity of diabetic neuropathy.

miR-199a-5p as a new therapeutic target for the treatment of idiopathic pulmonary fibrosis

Dr Bernard Mari is senior scientist in the Physiological Genomics group at the Institute of Molecular and Cellular Pharmacology (part of CNRS) in France. His research focuses on identifying therapeutic targets for idiopathic pulmonary fibrosis, a disease for which there is no current treatment apart from lung transplantation. The proposed project, performed in close collaboration with Dr Nicolas Pottier (University of Lille, France), aims to establish a preclinical proof of concept for the use of inhibitors of miR-199a-5p as well as specific Target Site Blockers for the interaction of miR-199a-5p with its target CAV1, to delay the progression of lung fibrosis in a mouse model.

SNALP-mediated miR-21 silencing: a novel microRNA-based gene therapy approach for glioblastoma

Dr. Maria Conceição Pedroso de Lima is Professor in the Department of Life Sciences and Head of the Vectors and Gene Therapy Group at the Center for Neuroscience and Cell Biology, University of Coimbra, Portugal. Her research is focused on the development of lipid-based gene delivery systems for application in gene therapy and her lab has been investigating the therapeutic potential of microRNAs for glioblastoma (GBM). The main goal of this project is to test a new multimodal gene therapy approach based on the use of tumor targeting SNALPs (stable nucleic acid lipid particles) encapsulating antimiR-21 oligonucleotides in combination with Sunitinib (a multiple tyrosine kinase inhibitor) in GBM-bearing mice.


Dr. Clare Rebbeck, Cold Spring Harbor Laboratory, NY, USA

Project title: miRNAs in the good vs the bad: myeloid cells in the tumor environment
Abstract: How a cancer evolves and progresses within an individual is governed by many factors, not only the errant tumor cells them selves but also the surrounding and the infiltrating cells. Myeloid cells are known to function as pro – tumor and anti-tumor depending on how the cell is activated. We plan to investigate how miRNAs are involved in this decision.

Dr. Charles Vanderburg, Harvard Medical School, Boston, USA

Project title: CSF-derived miRNA profiling as a biomarker of Neurodegenerative Diseases
Abstract: Human Cerebrospinal fluid contains miRNAs which are stabilized due to their sequestration within exosomes. This has allowed us to generate disease-specific profiles of CSF-derived miRNAs for Alzheimer’s disease, Parkinson’s disease and Amyotrophic Lateral Sclerosis. We plan to use Exiqon’s suite of miRNA tools to establish well-validated biomarkers for these neurodegenerative diseases.

Dr. Sara Larriba, Human Molecular Genetics Group, Bellvitge Biomedical Research Institute (IDIBELL), Barcelona, Spain

Project title: Profiling miRNA expression of spermatozoa in male infertility: Defining the fertility potential of sperm
Abstract: Recent evidences have shown that sperm transcriptome could reflect the fertilizing quality of spermatozoa; however the role of miRNAs in the regulation of spermatozoa gene expression has yet to be determined. We aim to identify miRNAs differentially expressed in male infertility of idiopathic origin contributing to the task of defining potential genetic markers for predicting the fecundity ability of spermatozoa.

Dr. Muthuswamy Balasubramanyam, Madras Diabetes Research Foundation, India

Project title: Exploring ‘UrinomiRs’ as biomarker of diabetic nephropathy
Abstract: There is an unmet clinical need to discover biomarkers other than microalbuminuria in predicting the development of nephropathy in patients with diabetes. miRNA signatures from body fluids have been shown associated not only with the disease but also the severity of disease. Our project aims to harness the power of the miRNA profiling to explore the biomarker potential of urine-based miRNAs (‘UrinomiRs’) in predicting the development of diabetic nephropathy.

Dr. Johannes Grillari, BOKU, Vienna, Austria

Project title: Serum microRNAs as biomarkers for osteoporosis
Abstract: So far no serum based biomarker has reached clinical application, and the currently best accepted diagnostic method is expensive and time consuming analysis of bone mineral density. Using Exiqon miRNA qPCR arrays we will therefore test levels of miRNAs in serum of osteoporosis patients versus age matched healthy controls. At the end we will have proof-of-principle if miRNAs are easy accessible novel biomarkers of osteoporosis and frailty.

Dr. Serena Lucotti, Institute of Clinical Physiology, CNR, Pisa, Italy

Project title: Circulating miRNAs as novel biomarkers of the efficacy of chemotherapeutic drugs
Abstract: Circulating miRNAs may represent potential indicators of the therapeutic efficacy of a drug. We plan to analyze the expression profile of extracellular miRNAs in the growth medium of prostate cancer cells treated with an antiproliferative drug. Our goal is to verify whether cytotoxicity correlates with the release of specific miRNAs and to clarify if the extracellular miRNAs play a role in cell-to-cell communication.

Dr. Viviana Bumaschny, INGEBI (CONICET) and University of Buenos Aires, Argentina

Project title: Identification of miRNAs involved in hypothalamic energy balance control
Abstract: Obesity is a chronic metabolic disorder affecting almost half a billion people that predisposes to type II diabetes and cardiovascular diseases. The hypothalamus regulates body energy balance by controlling food intake and energy expenditure. The general purpose of this study is to identify hypothalamic miRNAs that control genes involved in energy balance and to elucidate their normal physiological roles and participation in the etiology and perpetuation of obesity.

Dr. Richard Keijzer, University of Manitoba and Manitoba Institute of Child Health, Canada

Project title: MicroRNAs and nanoparticles: exploring a prenatal therapeutic intervention for congenital diaphragmatic hernia (CDH).
Abstract: Babies with CDH have abnormal diaphragm and lung development resulting in a high mortality and morbidity. The pathogenesis is poorly understood, but our pilot studies show that microRNAs play an important role. Improved prenatal detection has resulted in an opportunity to modulate the natural outcome. This proposal aims to explore the use of nanoparticles to deliver microRNAs as a new prenatal therapeutic intervention for pulmonary hypoplasia and CDH.

Dr. Carolyn Klinge, University of Louisville School of Medicine, Louisville, KY, USA

Project title : Dehydroepiandrosterone regulates microRNA expression in breast cancer cells
Abstract : miRNAs are dysregulated in breast cancer. Dehydroepiandrosterone (DHEA) is an important precursor for testosterone and estrogens in postmenopausal women and is also used as an over-the-counter anti-aging remedy. No one has examined how DHEA regulates miRNAs in breast cancer. We will identify miRNAs regulated by DHEA in human breast cancer cells and compare these results to estradiol-regulated miRNAs in the same cells.

Dr. Hanjoong Jo, Emory University, GA, USA

Project title :To identify the functional role of flow-sensitive miRNAs in atherosclerosis
Abstract : Atherosclerosis preferentially occurs at areas of disturbed flow in branched or curved arteries. In contrast, arterial regions that are exposed to stable flow are well-protected from atherosclerosis. Expression of miRNAs in endothelial cells are regulated by different flow conditions. We aim to determine the mechanisms by which flow-sensitive miRNAs regulate atherosclerosis by using LNA-based anti-miRNAs.



Dr. Helen Scott, University of Bristol, Bristol, United Kingdom

Project title: Investigating the roles of miRNAs in synaptic plasticity and learning and memory
Abstract: It is widely recognised that long-term plasticity and memory require changes in the proteome, however the role of miRNAs in the regulation of protein expression during these processes is only just beginning to be studied. We aim to identify miRNAs involved in the acquisition and consolidation of recognition memory and identify targets of these miRNAs.

Dr. Erick Tatro, UCSD, San Diego, USA

Project title: Targeting neurons to understand the microRNA framework in the brain
Abstract: We are using a rationally designed ligand-based system to deliver locked nucleic acids to specific neurons for altering microRNA function. The goal is to understand how microRNAs regulate dopamine homeostasis and reverse chemically-induced neuronal insults.

Dr. F. Gregory Wulczyn, Charite - Universitaetsmedizin Berlin, Berlin, Germany

Project title: Building the brain: Learning to read the miRNA blueprint
Abstract: miRNAs were discovered as timekeepers, genes that keep developmental events on schedule. We hope to acquire as comprehensive a view as possible of the role of miRNAs in temporal control of the brain’s architecture. We will first describe and then manipulate miRNA expression to reveal new features in the design of the mammalian cortex. 


Dr. Dipanjan Chowdhury, Harvard Medical School, Boston, USA

Project title: Developing a novel biochemical strategy to identify microRNAs targeting a specific transcript in a cellular context
Abstract: For most researchers investigating different biological problems, the key requirement is a reproducible strategy to identify microRNAs that target their gene of interest. We have developed a biochemical strategy of co-immunoprecipitating microRNA/mRNA interactions to identify physiologically relevant associations, and identify microRNAs that target a transcript in a cellular context.

Dr. Jaekwang Kim, Washington University, St. Louis, USA

Project title: Probing the roles of microRNAs in neurodegenerative diseases
Abstract: Alzheimer’s disease (AD) is the most common cause of dementia in the elderly but currently there is no effective therapy. MicroRNAs opened new era in the regulation of gene expression. We are interested in identifying novel microRNAs that modulate AD pathogenesis by screening microRNAs with miRCURY LNA miRNA inhibitors. We hope that this approach will lead us to find new therapeutic candidates for this devastating disease.

Mr. Himawan Harryanto, Dr. Kathy Gatford, Dr. Miles De Blasio, Ms. Patricia Grant, Prof. Julie Owens, University of Adelaide, Adelaide, Australia

Project title: Profiling microRNA Expression in the Pancreas of Placentally Restricted Young Adult Sheep Offspring
Abstract: Intra-uterine growth restriction (IUGR), due to reduced oxygen and nutrients before birth, causes diabetes in later life, partly due to reduced insulin secretory capacity and defects in the endocrine pancreas. This study will see if microRNA expression in the pancreas of adult IUGR sheep contributes to their poor insulin secretion.

Dr. Nicolas Leuenberger, Swiss Laboratory for Doping Analyses, Epalinges, Switzerland

Project title: Circulating microRNAs as powerful long-term biomarkers in Anti-Doping field
Abstract: The development of a minimally invasive test for the detection of forbidden substances in blood could greatly help the fight against doping in general. Circulating miRNAs have been demonstrated to be very specific and stable. Indeed, our preliminary results to detect blood doping lay the foundation for the development of miRNAs as a novel class of blood-based doping biomarkers.

Dr. Marcel Scheideler, Graz University of Technology, Graz, Austria

Project title: To combat overweight, obesity, and diabetes: Novel mechanisms with impact on white/brown fat cell balance
Abstract: Redirecting fat metabolism from energy storage to energy expenditure is a novel and promising strategy to fight overweight, obesity, and diabetes. We aim at identifying novel molecular mechanisms that are involved in the shift from energy storing white to energy dissipating brown adipocytes.

Dr. Victoria Ho, UCLA, Los Angeles, USA

Project title : The role of microRNAs in regulating local translation in neurons
Abstract : The neuron is a highly polarized cell type with special requirements for restricted gene expression. One way this restriction is achieved is by mRNA localization and regulated translation, possibly by microRNAs. My project aims to study the role of microRNAs in local translation by manipulating and measuring their levels in neurons, and by visualizing their spatial distribution.

Exiqon would like to thank all the participants of the program and would like to congratulate the winners. We hope to be able to award new grants through our grant program in the future.



Your local representative can answer any further questions you may have about the Grant program or Exiqon's products and services. Contact Exiqon to learn more .

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