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New Perspectives for Gene Therapy in Endocrinology
European Journal of Endocrinology (2000) 143 447±466 ISSN 0804-4643 INVITED REVIEW New perspectives for gene therapy in endocrinology Luisa Barzon, Roberta Bonaguro1, Giorgio PaluÁ 1 and Marco Boscaro Department of Medical and Surgical Sciences and 1Department of Histology, Microbiology and Medical Biotechnologies, University of Padova, Padova, Italy (Correspondence should be addressed to M Boscaro, Department of Medical and Surgical Sciences, University of Padova, Via Ospedale, 105, 35128 Padova, Italy; Email: [email protected], or to G PaluÁ, Department of Histology, Microbiology and Medical Biotechnologies, University of Padova, Via A Gabelli, 63, 35121 Padova, Italy; Email: [email protected]) Abstract Gene therapy for endocrine diseases represents an exciting new type of molecular intervention that may be a curative one. Endocrine disorders that might be treated by gene therapy include monogenic diseases, such as GH de®ciency and hypothalamic diabetes insipidus, and multifactorial diseases, such as diabetes mellitus, obesity and cancer. Premises seem promising for endocrine tumours, but many combined approaches of cell and gene therapy are foreseeable also for other endocrine disorders. This review outlines the principles of gene therapy, describes the endocrine disorders that might take advantage of gene transfer approaches, as well as the gene therapy interventions that have already been attempted, their major limitations and the problems that remain to be solved. European Journal of Endocrinology 143 447±466 Introduction in 1989 (data from Journal of Gene Medicine, www. wiley.co.uk/genmed, updated to 1 September 1999) (1). In the last three decades scienti®c progress in biomedi- Even though the disorders targeted by gene therapy cal research has revealed the molecular mechanisms approaches span the entire spectrum of human and genetic bases of many human diseases. -
How to Improve the Management of Acute Ischemic Stroke by Modern Technologies, Artificial Intelligence, and New Treatment Methods
life Review How to Improve the Management of Acute Ischemic Stroke by Modern Technologies, Artificial Intelligence, and New Treatment Methods Kamil Zele ˇnák 1,2,* , Antonín Krajina 3, Lukas Meyer 4, Jens Fiehler 4, ESMINT Artificial Intelligence and Robotics Ad hoc Committee 2, Daniel Behme 2,5, Deniz Bulja 2,6, Jildaz Caroff 2,7 , Amar Ajay Chotai 2,8 , Valerio Da Ros 2,9 , Jean-Christophe Gentric 2,10, Jeremy Hofmeister 2,11, Omar Kass-Hout 2,12 , Özcan Kocatürk 2,13, Jeremy Lynch 2,14, Ernesto Pearson 2,15 and Ivan Vukasinovic 2,16 1 Clinic of Radiology, Jessenius Faculty of Medicine in Martin, Comenius University in Bratislava, 03659 Martin, Slovakia 2 ESMINT Artificial Intelligence and Robotics Ad hoc Committee, ESMINT, 8008 Zurich, Switzerland; offi[email protected] (E.A.I.R.A.h.C.); [email protected] (D.B.); [email protected] (D.B.); [email protected] (J.C.); [email protected] (A.A.C.); [email protected] (V.D.R.); [email protected] (J.-C.G.); [email protected] (J.H.); [email protected] (O.K.-H.); [email protected] (Ö.K.); [email protected] (J.L.); [email protected] (E.P.); [email protected] (I.V.) 3 Citation: Zeleˇnák,K.; Krajina, A.; Department of Radiology, Charles University Faculty of Medicine and University Hospital, CZ-500 05 Hradec Králové, Czech Republic; [email protected] Meyer, L.; Fiehler, J.; ESMINT 4 Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Artificial Intelligence and Robotics 20251 Hamburg, Germany; [email protected] (L.M.); fi[email protected] (J.F.) Ad hoc Committee; Behme, D.; Bulja, 5 University Clinic for Neuroradiology, Medical Faculty, Otto-von-Guericke University Magdeburg, D.; Caroff, J.; Chotai, A.A.; Da Ros, V.; 39120 Magdeburg, Germany et al. -
PROGRESS in NEUROSCIENCE PINS “CNS Lymphatic Drainage In
PROGRESS IN NEUROSCIENCE PINS Seminar Series of the Brain & Mind Research Institute Weill Cornell Medical College (WCMC) & The Graduate Program in Neuroscience of WCMC and Sloan Kettering Institute Thursday, 12/8/16, 4 PM, coffee at 3:45 PM Uris Auditorium “CNS lymphatic drainage in health and disease” Jonathan Kipnis, Ph.D., Center for Brain Immunology and Glia (BIG), Harrison Distinguished Teaching Professor of Neuroscience and Chair, Department of Neuroscience, University of Virginia Abstract The central nervous system was considered to be devoid of classical lymphatic drainage. We recently challenged that paradigm by demonstrating the presence of a lymphatic vasculature in the surrounding of the brain called the meninges. We demonstrated that lymphatic vessels, expressing the markers for lymphatic endothelial cells (LEC; i.e Lyve-1, Prox1, podoplanin, VEGFR3 and CCL21) are located along the dural sinuses. They present features of initial lymphatics, and, importantly, drain fluids, macromolecules and immune cells from the cerebrospinal fluid and the CNS parenchyma into the deep cervical lymph nodes. Our recent efforts are concentrated on understanding the role of meningeal lymphatic vessels in CNS function in health and disease. Our results suggest that the drainage into the deep cervical lymph nodes might play different roles at different stages of several neurological diseases. Understanding the function of the lymphatic drainage in CNS might shed a new light on neurological disorders and offer new therapeutic targets. Recent Relevant Publications: 1. Gadani SP, Walsh JT, Smirnov I, Zheng J and Kipnis J. (2015) The glia-derived alarmin IL-33 orchestrates the post CNS injury immune response and promotes recovery. -
Quarterly Report: Second Quarter 2017
QUARTERLY REPORT: 2ND QUARTER 2017 Q2 2017 Research Consortium Welcomes Three New Members The Brain’s 2 Phyllis Rappaport Updates Former College Classmates Lymphatic System 3 Modern science has an incredibly thorough understanding of the human body. It is J. McLaughlin’s ‘Sip ’n Shop’ hard to imagine that any organ or system could exist within the body that has yet to be Fundraiser discovered. Yet this is exactly what happened in 2015, when researchers discovered 4 lymphatic vessels around and within the brain. Living with Alzheimer’s Film The lymphatic system exists throughout the body. Part of the immune system, it consists Screening of a network of channels (vessels) and glands called “nodes.” Nodes create immune cells 5 to help the body fight infection. The vessels carry fluid containing these immune cells, as well as pathogens and harmful cellular waste products, away from organs. Until recently, CaringKind Alliance Update lymphatic vessels had never been observed in or around the brain. While there were other 5 known ways—such as immune cells called macrophages—for waste products to be Extra Sets of Hands cleared from the brain, researchers had trouble explaining the volume of clearance they observed without a lymphatic system. 5 Cure Alzheimer’s Fund A recent study at the University of Virginia by Jonathan Kipnis, Ph.D., and his colleagues Heroes Antoine Louveau, Ph.D., and Tajie Harris, Ph.D., shed new light on this problem. Using a new method of examining the meninges, a membrane that covers the brain, they 6 discovered there was in fact a network of lymphatic vessels surrounding the brain. -
Recombinant Dna Advisory Committee
RECOMBINANT DNA ADVISORY COMMITTEE Minutes of Meeting March 7 and 8, 2012 U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICES Public Health Service National Institutes of Health Minutes of the Recombinant DNA Advisory Committee, 3/7–8/12 Contents I. Call to Order and Opening Remarks ................................................................................................... 2 II. Review and Discussion of Human Gene Transfer Protocol #1201-1144 titled: AAV8-Mediated Low Density Lipoprotein Receptor Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia .......................................................................................................................... 2 A. Protocol Summary ........................................................................................................................ 2 B. Written Reviews by RAC Members .............................................................................................. 3 C. RAC Discussion ............................................................................................................................ 4 D. Investigator Response .................................................................................................................. 4 1. Written Responses to RAC Reviews ..................................................................................... 4 2. Responses to RAC Discussion Questions ............................................................................. 6 E. Public Comment ........................................................................................................................... -
A Strategic Investment in Pediatrics and Infectious Diseases Dr
December 2020 milestones Dr. Sallie Permar, the new chair of Weill Cornell Medicine’s Department of Pediatrics A Strategic Investment in Pediatrics and Infectious Diseases Dr. Sallie Permar, a distinguished physician-scientist who of such viruses as HIV, Zika and cytomegalovirus (CMV), the specializes in pediatric infectious diseases, joined Weill Cornell most common congenital infection and a leading cause of birth Medicine on December 1 as the new chair of the Department defects. In her research, she also discovered a protein in breast of Pediatrics. milk that neutralizes HIV, the virus that causes AIDS. The recruitment of Dr. Permar is part of Weill Cornell Medicine’s “Dr. Permar will enhance our mission in both pediatrics and strategic investment in pediatrics and infectious disease research infectious diseases, building on our wealth of research as she and clinical care, with a goal of raising nearly $60 million to support collaborates with investigators and clinicians to improve the expanded translational research efforts in the Belfer Research lives of children,” says Dr. Augustine M.K. Choi, the Stephen and Building. The COVID-19 pandemic has reinforced the growing need Suzanne Weiss Dean. “As a leading academic medical center, for research of infectious diseases of all types – including areas in we must expand our investment in infectious diseases with an which Dr. Permar specializes. She and her team are working on the eye toward future global pathogens that can have a profound development of vaccines to prevent mother-to-child transmission impact on human health.” continued on page 2 A Strategic Investment in Pediatrics and Infectious Diseases continued from cover Infectious disease experts at Well Cornell Medicine more than $3 million to establish the Gerald M. -
2018 Winter Newsletter
Holiday Gift Ideas A Q&A wth Alan Arnette Plan Your Giving Now Celebrate the reason for the season Arnette is a mountaineer, speaker Make giving a commitment with a gift that gives back and Alzheimer’s advocate to Cure Alzheimer's Fund WINTER SEASON 2018 THE CASE FOR HOPE Panel Explores the Path Forward in Alzheimer’s Research at Our 8th Annual Scientific Symposium THE CASE FOR HOPE SCIENCE PANEL EXPLORES THE PATH FORWARD IN ALZHEIMER’S RESEARCH The 8th Annual Cure Alzheimer’s Fund Symposium featured award-winning NPR science writer Jon Hamilton moderating a discussion between Drs. Ron Petersen, Bob Vassar and Teresa Gomez-Isla on the Case For Hope in Alzheimer’s disease research. Co-Chairman Jeff Morby started the meeting by announcing that for the frst time. She remembered being impacted by learning Cure Alzheimer’s Fund had just received the designation of Top that the disease can steal one of the “greatest treasures we have 10 Best Medical Research Organizations by nonproft watchdog as human beings—our memories.” Charity Navigator. After providing the audience with an update of CureAlz's results, including $75 million distributed for 340 Progressing Through Challenges research grants to 127 of the world’s leading researchers, he then introduced Dr. Rudy Tanzi, who introduced the panel: Dr. Petersen The panelists described the challenge of connecting the events as a champion for scientists whose policy work has been pav- happening in the brain to the symptoms experienced by patients. ing the way for clinical trials that target amyloid before the onset All three of the scientists explained that amyloid plaques and of symptoms, Dr. -
Embryonic Stem Cells Modulate the Cancer-Permissive
Theranostics 2019, Vol. 9, Issue 16 4764 Ivyspring International Publisher Theranostics 2019; 9(16): 4764-4778. doi: 10.7150/thno.33139 Research Paper Embryonic Stem Cells Modulate the Cancer-Permissive Microenvironment of Human Uveal Melanoma Jiahui Liu1*, Zheqian Huang1*, Liu Yang1, Xiaoran Wang1, Shoubi Wang1, Chaoyang Li1, Ying Liu1, Yaqi Cheng1, Bowen Wang1, Xuan Sang1, Xiongjun He1, Chenjie Wang1, Tengfei Liu1, ChengXiu Liu2, Lin Jin1, Chang Liu1, Xiaoran Zhang3, Linghua Wang4, Zhichong Wang1 1. State Key Laboratory of Ophthalmology, Zhongshan Ophthalmic Center, Sun Yat-sen University, Guangzhou 510060, P. R. China. 2. Department of Ophthalmology, Affiliated Hospital of Qingdao University Medical College, Qingdao 266000, P. R. China 3. Center for Stem Cell Biology and Tissue Engineering, Key Laboratory for Stem Cells and Tissue Engineering, Ministry of Education, Sun Yat-Sen University, Guangzhou 510275, P. R. China 4. Department of Genomic Medicine, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas 77030, USA. *These authors contributed equally to this work. Corresponding author: [email protected] © The author(s). This is an open access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/). See http://ivyspring.com/terms for full terms and conditions. Received: 2019.01.14; Accepted: 2019.05.14; Published: 2019.07.09 Abstract The currently used anti-cancer therapies work by killing cancer cells but result in adverse effects and resistance to treatment, which accelerates aging and causes damage to normal somatic cells. On one hand, chicken and zebrafish embryos can reprogram cancer cells towards a non-tumorigenic phenotype; however, they cannot be used in the clinical practice. -
2020 Annual Report Our Mission
2020 ANNUAL REPORT OUR MISSION Cure Alzheimer’s Fund is a nonprofit organization dedicated to funding research with the highest probability of preventing, slowing or reversing Alzheimer’s disease. Annual Report 2020 MESSAGE FROM THE CHAIRMEN 2 THE MAIN ELEMENTS OF THE PATHOLOGY OF ALZHEIMER’S DISEASE 9 RESEARCH AREAS OF FOCUS 10 PUBLISHED PAPERS 12 CURE ALZHEIMER’S FUND CONSORTIA 20 OUR RESEARCHERS 22 2020 FUNDED RESEARCH 32 2020 EVENTS TO FACILITATE RESEARCH COLLABORATION 68 MESSAGE FROM THE PRESIDENT 70 2020 FUNDRAISING 72 2020 FINANCIALS 73 OUR PEOPLE 74 OUR HEROES 75 AWARENESS 78 IN MEMORY AND IN HONOR 80 SUPPORT OUR RESEARCH 81 Message From The Chairmen Dear Friends, 2020 was a truly remarkable year: • Despite the COVID-19 pandemic, we were fully functional with all of our wonderful staff working from their homes. We were able to pay and retain all of our employees, thanks to the generosity of our directors. • And, amazingly, we were able to increase our fundraising by 2%, in this very tough year, to $25.9 million provided by 21,000 donors. • The above enabled us to fund 59 research grants totaling $16.5 million. We have, since inception, financed 525 grants, representing $125 million in cumulative funding through March 2021. • We have one therapy well on its way through clinical trials, and another expected to enter clinical trials in late 2021 or 2022. Our Scientists Approximately 175 scientists affiliated with 75 institutions around the world are working on our projects. They are profiled in the pages that follow. Many labs faced funding challenges during COVID-19, and our consistent support was very beneficial for ensuring that vital staff could be retained and scientific progress was preserved. -
University of Copenhagen, Copenhagen, Denmark
Understanding the functions and relationships of the glymphatic system and meningeal lymphatics Louveau, Antoine; Plog, Benjamin A.; Antila, Salli; Alitalo, Kari; Nedergaard, Maiken; Kipnis, Jonathan Published in: The Journal of Clinical Investigation DOI: 10.1172/JCI90603 Publication date: 2017 Document version Publisher's PDF, also known as Version of record Document license: Unspecified Citation for published version (APA): Louveau, A., Plog, B. A., Antila, S., Alitalo, K., Nedergaard, M., & Kipnis, J. (2017). Understanding the functions and relationships of the glymphatic system and meningeal lymphatics. The Journal of Clinical Investigation, 127(9), 3210-3219. https://doi.org/10.1172/JCI90603 Download date: 26. Sep. 2021 Understanding the functions and relationships of the glymphatic system and meningeal lymphatics Antoine Louveau, … , Maiken Nedergaard, Jonathan Kipnis J Clin Invest. 2017;127(9):3210-3219. https://doi.org/10.1172/JCI90603. Review Series Recent discoveries of the glymphatic system and of meningeal lymphatic vessels have generated a lot of excitement, along with some degree of skepticism. Here, we summarize the state of the field and point out the gaps of knowledge that should be filled through further research. We discuss the glymphatic system as a system that allows CNS perfusion by the cerebrospinal fluid (CSF) and interstitial fluid (ISF). We also describe the recently characterized meningeal lymphatic vessels and their role in drainage of the brain ISF, CSF, CNS-derived molecules, and immune cells from the CNS and meninges to the peripheral (CNS-draining) lymph nodes. We speculate on the relationship between the two systems and their malfunction that may underlie some neurological diseases. Although much remains to be investigated, these new discoveries have changed our understanding of mechanisms underlying CNS immune privilege and CNS drainage. -
MISTIC Collaborative Stroke Update
MISTIC Collaborative Stroke Update Friday, April 30, 2021 12 p.m. – 4 p.m. and Saturday, May 1, 2021 8 a.m. – 12 p.m. Virtual Presentation via Zoom According to the Centers of Disease Control and Prevention, stroke afflicts nearly 800,000 people and causes approximately 130,000 deaths in the US each year. Stroke is also the leading cause of long-term disability and costs our nation billions in treatment dollars on an annual basis, which causes significant challenges for physicians and allied healthcare professionals. Through the use of lectures and case discussion, national and regional experts will address the latest advances in pre-hospital stroke care, treatment of intracranial aneurysms, extracranial and intracranial atherosclerotic disease and techniques of mechanical thrombectomy. The purpose of this conference is to provide neurosurgeons, neurologists, family medicine, internal medicine, trauma and emergency room physicians, residents, advanced practice providers, nurses, pharmacists, radiology techs, and other healthcare professionals with advances in stroke care. The MISTIC Mission: The MIchigan Stroke Treatment Improvement Collaborative (MISTIC) is a multi-institutional effort led by cerebrovascular specialists across greater Michigan and northwest Ohio areas to improve stroke care, advance clinical research, and train the next generation of clinicians. Participating Institutions: Michigan Medicine, ProMedica, University of Toledo, Ascension St. John/Providence Park, Ascension St. Mary’s Saginaw, Beaumont Royal Oak, DMC, Henry Ford Hospital, McLaren Flint/Macomb, Metro Health, Munson Medical Center, Sparrow, Spectrum, St. Vincent Mercy Presented by ProMedica’s Continuing Medical Education Department Faculty Joseph Adel, MD, FAANS Cerebrovascular, Endovascular & Skull Base Neurosurgery Clinical Associate Professor, Central Michigan University Ascension St. -
Bone Marrow Transplant Alleviates Rett Symptoms in Mice
Spectrum | Autism Research News https://www.spectrumnews.org NEWS Bone marrow transplant alleviates Rett symptoms in mice BY EMILY SINGER 19 MARCH 2012 Managing microglia: Mutant mice lacking the MeCP2 protein are small (left), while those bred to have a normal version of the MeCP2 protein in their myeloid cells ? precursors to microglia ? grow to normal size (right). A bone marrow transplant from healthy mice to those lacking the MeCP2 protein, which causes Rett syndrome in humans when mutated, extends lifespan and alleviates symptoms of the disorder, according to research published online 18 March in Nature1. Researchers found that microglia, which take root in the brain after the transplant, are the most important component of the treatment. These cells are known to act as scavengers that clean up cellular debris, but recent research suggests they also have other roles, including influencing neural transmission and development of the synapse, the junction between neurons. Rett syndrome is a rare disorder caused by deletion or mutation of the X-linked MeCP2 gene and occurs almost exclusively in females. Symptoms emerge around 6 to 18 months of age and include breathing problems, intellectual disability, seizures, language deficits and other autism-like impairments. Mice in which the MeCP2 gene has been knocked out in all or a subset of cells mimic many of the features of Rett syndrome. 1 / 4 Spectrum | Autism Research News https://www.spectrumnews.org Following treatment, abnormal breathing patterns and apnea, a temporary interruption in breathing seen in both MeCP2 knockout mice and children with Rett syndrome, are diminished in the knockout animals.