DOCSLIB.ORG

Stem Cell and Gene-Based Therapy Alexander Battler and Jonathan Leor Stem Cell and Gene-Based Therapy Frontiers in Regenerative Medicine

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
Stem Cell and Gene-Based Therapy Alexander Battler and Jonathan Leor Stem Cell and Gene-Based Therapy Frontiers in Regenerative Medicine Stem Cell and Gene-Based Therapy Alexander Battler and Jonathan Leor Stem Cell and Gene-Based Therapy Frontiers in Regenerative Medicine With 62 Figures including 27 Color Plates Alexander Battler, MD, FACC, FESC Jonathan Leor, MD, FACC, FESC Department of Cardiology Neufeld Cardiac Research Institute Rabin Medical Center, Petach-Tikwa Sackler Faculty of Medicine Sackler Faculty of Medicine Tel Aviv University Tel Aviv University Sheba Medical Center Israel Tel-Hashomer Israel British Library Cataloguing in Publication Data Stem cell and gene-based therapy: frontiers in regenerative medicine 1. Stem cells – Research 2. Gene therapy – Research 3. Regeneration (Biology) – Research I. Battler, Alexander II. Leor, Jonathan 616′.02774 ISBN-10: 1852339799 Library of Congress Control Number: 2005925974 ISBN-10: 1-85233-979-9 e-ISBN 1-84628-142-3 Printed on acid-free paper ISBN-13: 978-1-85233-979-1 © Springer-Verlag London Limited 2006 Apart from any fair dealing for the purposes of research or private study, or criticism or review, as permitted under the Copyright, Designs and Patents Act 1988, this publication may only be reproduced, stored or trans- mitted, in any form or by any means, with the prior permission in writing of the publishers, or in the case of reprographic reproduction in accordance with the terms of licences issued by the Copyright Licensing Agency. Enquiries concerning reproduction outside those terms should be sent to the publishers. The use of registered names, trademarks, etc. in this publication does not imply, even in the absence of a spe- cific statement, that such names are exempt from the relevant laws and regulations and therefore free for gen- eral use. Product liability: The publisher can give no guarantee for information about drug dosage and application thereof contained in this book. In every individual case the respective user must check its accuracy by con- sulting other pharmaceutical literature. Printed in the United States of America (SPI/SB) 987654321 Springer Science+Business Media springeronline.com Foreword Regenerative medicine promises to be one of the great future frontiers. Critical to the success of this area is stem cell research, and cell- and gene-based therapy. In this book, a variety of outstanding authors discuss important aspects of these research areas. Important topics include new types of cell therapies and stem cell-based therapy for angiogenesis and cardiac repair. They also include cell-therapy approaches in neuro- logic areas such as Parkinson’s disease, multiple sclerosis, and stroke. A third area that is explored involves the development of cell-based systems for cartilage and bone repair, bladder repair, and kidney regeneration. A fourth area involves important work in the eye, specifically, optic nerve regeneration, retinal repair, and ocular surface regenera- tion. The potential of using insulin-producing cells or islets is also examined in this book, as are strategies for cord blood transplantation for various hematologic disorders. Finally, important areas of research involving skin regeneration and wound healing are discussed. This book should provide an excellent overview of the important areas in regenerative medicine with respect to cell and gene therapy and hopefully will be a use- ful guide for scientists doing research in these areas. Robert Langer Massachusetts Institute of Technology Cambridge, MA, USA v Preface The human body has limited potential to rejuvenate injured organs and tissues. An old dream of scientists and physicians is to be able to rebuild “spare parts” to replace injured or diseased tissues—a notion that was once referred to as the field of science fic- tion. The new discipline of regenerative medicine aims to help the body heal itself with cells, genes, and bioactive molecules and materials. In the last decade, the notion that stem cells can give rise to mature tissues has made stem cells the focus of intense research designed to explore their promise for the treatment of a variety of diseases. The aim of our book is to cover key aspects of the promise and existing problems in the emerging field of regenerative medicine. With the contribution of leading figures and pioneers in various disciplines of regenerative medicine and science, the book brings together major approaches of stem cell and gene-based therapy in one text. The appearance of this book has been made by the willing and corporation of many individuals. We thank our contributors, and section editors Belkin, Dekel, Efrat, Grossman, Melamed, Nagler, Offen, Nevo, and Reisner for generously sharing their expertise and scientific skills on which this book is based. We hope that the book will provide a realistic image of the huge potential, promise and challenges facing the fan- tastic field of regenerative medicine in its quest to cure disease and prolong life. Alexander Battler Jonathan Leor vii Acknowledgment We thank the section editors and coauthors for generously sharing their expertise and skills on which this book is based. We thank Mrs. Elaine Finkelstein from Neufeld Cardiac Research Institute, Tel-Hashomer, Israel, for her unfailing skills that were very helpful in editing and preparing this book. Alexander Battler, MD, FACC, FESC Jonathan Leor, MD, FACC, FESC ix Contents Foreword Robert Langer . v Preface Alexander Battler and Jonathan Leor . vii Acknowledgment . ix List of Contributors . xv Section 1 Heart Introduction Alexander Battler and Jonathan Leor . 2 1 Renovation of the Injured Heart with Myocardial Tissue Engineering Jonathan Leor, Natali Landa, and Smadar Cohen . 3 2 Adult Stem Cells for Myocardial Tissue Repair Dirk Strunk and Christof Stamm . 17 3 Regeneration of the Functional Myocardium Using Human Embryonic Stem Cells Oren Caspi and Lior Gepstien . 33 4 Therapeutic Angiogenesis Shmuel Fuchs and Alexander Battler . 45 5 Cell Therapy for Heart Failure Thorsten Reffelmann and Robert A. Kloner . 59 Section 2 Neuro Introduction Daniel Offen and Eldad Melamed . 72 6 Cell Transplantation for Diseases of Myelin Tamir Ben-Hur and Ofira Einstein . 75 xi xii Contents 7 Stem Cells as a Source for Cell Replacement in Parkinson’s Disease Daniel Offen, Yossef S. Levy, and Eldad Melamed . 97 8 Cell Replacement Therapy in Acute Stroke: Current State Yossi Gilgun-Sherki and Jonathan Y. Streifler . 123 9 Gene Therapy to the Nervous System Hillel Haim and Israel Steiner . 133 Section 3 Musculoskeletal Introduction Zvi Nevo and Mark M. Levy . 156 10 Mesenchymal Stem Cells: Where Can You Find Them? How Can You Use Them? Anna Derubeis, Giuseppina Pennesi, and Ranieri Cancedda . 159 11 Basic to Clinical Cartilage Engineering: Past, Present, and Future Discussions Mats Brittberg, Tommi Tallheden, and Anders H. Lindahl . 169 12 Cartilage Rocky S. Tuan and Faye H. Chen . 179 13 Bone Regeneration A.H. Reddi . 195 14 Osteoarthritis and Mesenchymal Cells – The Prospects for Repair of the Disease by Cell Transplantation and Tissue Engineering Dror Robinson . 203 Section 4 Kidney Introduction Benjamin Dekel and Yair Reisner . 208 15 Progenitor Cell Therapy for Kidney Regeneration Benjamin Dekel and Yair Reisner . 209 16 Tissue Engineering – The Bladder Anthony Atala . 225 Section 5 Eye Introduction Michael Belkin . 234 17 Neuroprotection in Ophthalmology: A Review Yaniv Barkana and Michael Belkin . 237 Contents xiii 18 Autoimmunity for Central Nervous System Maintenance, Regeneration, and Renewal: Development of a T Cell-Based Vaccination Against Neurodegeneration Michal Schwartz and Jonathan Kipnis . 251 19 Retinal Repair by Stem Cell Transplantation Jeffrey H. Stern, Sally Temple, and Soma De . 259 20 Induction of Ocular Surface Regeneration Irina S. Barequet . 281 Section 6 Pancreas Introduction Shimon Efrat . 298 21 Insulin-Producing Cells Generated from Nonpancreatic Tissues Shimon Efrat . 301 22 Generation of Islets from Pancreatic Progenitor Cells Susan Bonner-Weir, Tandy Aye, Akari Inada, Elena Toschi, and Arun Sharma . 309 23 Embryonic Stem Cells as a Source of Pancreatic Precursors and Islet Cells In Vitro Victoria L. Browning, Brenda W. Kahan, and Jon S. Odorico . 321 Section 7 Hematology Introduction Arnon Nagler . 332 24 Human Umbilical Cord Blood Transplantation: A Viable Option for Stem Cell Graft Gal Goldstein, Amos Toren, and Arnon Nagler . 333 25 Nonmyeloablative Stem Cell Transplantation in the Treatment of Hematologic Malignancies Avichai Shimoni and Arnon Nagler . 351 26 Hematopoietic Stem Cell Transplantation from Human Leukocyte Antigen Haploidentical Donor Merav Leiba and Arnon Nagler . 361 Section 8 Skin Introduction Nili Grossman . 372 27 Wound Healing and Skin Substitutes Adam J. Singer and Marcia Simon . 375 xiv Contents 28 Skin Regeneration from Multipotent Adult and Embryonic Stem Cells Kursad Turksen and Tammy-Claire Troy . 395 Index . 407 Color Insert . following page 206 Contributors Anthony Atala, MD Susan Bonner-Weir, PhD The William Boyce Professor and Senior Investigator, Joslin Diabetes Chair, Department of Urology Center Director, Wake Forest Institute for Associate Professor of Medicine, Regenerative Medicine Harvard Medical School Wake Forest University School of Boston, MA, USA Medicine Winston-Salem, NC, USA Mats Brittberg, MD, PhD Cartilage Research Unit Irina S. Barequet, MD Göteborg University The Goldschleger Eye Institute Department of Orthopaedics Sheba Medical Center Sackler Kungsbacka Hospital Faculty of Medicine Kungsbacka, Sweden Tel Aviv University, Tel Aviv, Israel Ranieri Cancedda, MD Professor Alexander Battler, MD, FACC,
Load more

Recommended publications
  • ORIGINS of the PALESTINE MANDATE by Adam Garfinkle
    NOVEMBER 2014 ORIGINS OF THE PALESTINE MANDATE By Adam Garfinkle Adam Garfinkle, Editor of The American Interest Magazine, served as the principal speechwriter to Secretary of State Colin Powell. He has also been editor of The National Interest and has taught at Johns Hopkins University’s School for Advanced International Studies (SAIS), the University of Pennsylvania, Haverford College and other institutions of higher learning. An alumnus of FPRI, he currently serves on FPRI’s Board of Advisors. This essay is based on a lecture he delivered to FPRI’s Butcher History Institute on “Teaching about Israel and Palestine,” October 25-26, 2014. A link to the the videofiles of each lecture can be found here: http://www.fpri.org/events/2014/10/teaching-about- israel-and-palestine Like everything else historical, the Palestine Mandate has a history with a chronological beginning, a middle, and, in this case, an end. From a strictly legal point of view, that beginning was September 29, 1923, and the end was midnight, May 14, 1948, putting the middle expanse at just short of 25 years. But also like everything else historical, it is no simple matter to determine either how far back in the historical tapestry to go in search of origins, or how far to lean history into its consequences up to and speculatively beyond the present time. These decisions depend ultimately on the purposes of an historical inquiry and, whatever historical investigators may say, all such inquiries do have purposes, whether recognized, admitted, and articulated or not. A.J.P. Taylor’s famous insistence that historical analysis has no purpose other than enlightened storytelling, rendering the entire enterprise much closer to literature than to social science, is interesting precisely because it is such an outlier perspective among professional historians.
    [Show full text]
  • Most Common Jewish First Names in Israel Edwin D
    Names 39.2 (June 1991) Most Common Jewish First Names in Israel Edwin D. Lawson1 Abstract Samples of men's and women's names drawn from English language editions of Israeli telephone directories identify the most common names in current usage. These names, categorized into Biblical, Traditional, Modern Hebrew, and Non-Hebrew groups, indicate that for both men and women over 90 percent come from Hebrew, with the Bible accounting for over 70 percent of the male names and about 40 percent of the female. Pronunciation, meaning, and Bible citation (where appropriate) are given for each name. ***** The State of Israel represents a tremendous opportunity for names research. Immigrants from traditions and cultures as diverse as those of Yemen, India, Russia, and the United States have added their onomastic contributions to the already existing Jewish culture. The observer accustomed to familiar first names of American Jews is initially puzzled by the first names of Israelis. Some of them appear to be biblical, albeit strangely spelled; others appear very different. What are these names and what are their origins? Benzion Kaganoffhas given part of the answer (1-85). He describes the evolution of modern Jewish naming practices and has dealt specifi- cally with the change of names of Israeli immigrants. Many, perhaps most, of the Jews who went to Israel changed or modified either personal or family name or both as part of the formation of a new identity. However, not all immigrants changed their names. Names such as David, Michael, or Jacob required no change since they were already Hebrew names.
    [Show full text]
  • Aliyah and Settlement Process?
    Jewish Women in Pre-State Israel HBI SERIES ON JEWISH WOMEN Shulamit Reinharz, General Editor Joyce Antler, Associate Editor Sylvia Barack Fishman, Associate Editor The HBI Series on Jewish Women, created by the Hadassah-Brandeis Institute, pub- lishes a wide range of books by and about Jewish women in diverse contexts and time periods. Of interest to scholars and the educated public, the HBI Series on Jewish Women fills major gaps in Jewish Studies and in Women and Gender Studies as well as their intersection. For the complete list of books that are available in this series, please see www.upne.com and www.upne.com/series/BSJW.html. Ruth Kark, Margalit Shilo, and Galit Hasan-Rokem, editors, Jewish Women in Pre-State Israel: Life History, Politics, and Culture Tova Hartman, Feminism Encounters Traditional Judaism: Resistance and Accommodation Anne Lapidus Lerner, Eternally Eve: Images of Eve in the Hebrew Bible, Midrash, and Modern Jewish Poetry Margalit Shilo, Princess or Prisoner? Jewish Women in Jerusalem, 1840–1914 Marcia Falk, translator, The Song of Songs: Love Lyrics from the Bible Sylvia Barack Fishman, Double or Nothing? Jewish Families and Mixed Marriage Avraham Grossman, Pious and Rebellious: Jewish Women in Medieval Europe Iris Parush, Reading Jewish Women: Marginality and Modernization in Nineteenth-Century Eastern European Jewish Society Shulamit Reinharz and Mark A. Raider, editors, American Jewish Women and the Zionist Enterprise Tamar Ross, Expanding the Palace of Torah: Orthodoxy and Feminism Farideh Goldin, Wedding Song: Memoirs of an Iranian Jewish Woman Elizabeth Wyner Mark, editor, The Covenant of Circumcision: New Perspectives on an Ancient Jewish Rite Rochelle L.
    [Show full text]
  • Trend Analysis the Israeli Unit 8200 an OSINT-Based Study CSS
    CSS CYBER DEFENSE PROJECT Trend Analysis The Israeli Unit 8200 An OSINT-based study Zürich, December 2019 Risk and Resilience Team Center for Security Studies (CSS), ETH Zürich Trend analysis: The Israeli Unit 8200 – An OSINT-based study Author: Sean Cordey © 2019 Center for Security Studies (CSS), ETH Zurich Contact: Center for Security Studies Haldeneggsteig 4 ETH Zurich CH-8092 Zurich Switzerland Tel.: +41-44-632 40 25 [email protected] www.css.ethz.ch Analysis prepared by: Center for Security Studies (CSS), ETH Zurich ETH-CSS project management: Tim Prior, Head of the Risk and Resilience Research Group, Myriam Dunn Cavelty, Deputy Head for Research and Teaching; Andreas Wenger, Director of the CSS Disclaimer: The opinions presented in this study exclusively reflect the authors’ views. Please cite as: Cordey, S. (2019). Trend Analysis: The Israeli Unit 8200 – An OSINT-based study. Center for Security Studies (CSS), ETH Zürich. 1 Trend analysis: The Israeli Unit 8200 – An OSINT-based study . Table of Contents 1 Introduction 4 2 Historical Background 5 2.1 Pre-independence intelligence units 5 2.2 Post-independence unit: former capabilities, missions, mandate and techniques 5 2.3 The Yom Kippur War and its consequences 6 3 Operational Background 8 3.1 Unit mandate, activities and capabilities 8 3.2 Attributed and alleged operations 8 3.3 International efforts and cooperation 9 4 Organizational and Cultural Background 10 4.1 Organizational structure 10 Structure and sub-units 10 Infrastructure 11 4.2 Selection and training process 12 Attractiveness and motivation 12 Screening process 12 Selection process 13 Training process 13 Service, reserve and alumni 14 4.3 Internal culture 14 5 Discussion and Analysis 16 5.1 Strengths 16 5.2 Weaknesses 17 6 Conclusion and Recommendations 18 7 Glossary 20 8 Abbreviations 20 9 Bibliography 21 2 Trend analysis: The Israeli Unit 8200 – An OSINT-based study selection tests comprise a psychometric test, rigorous Executive Summary interviews, and an education/skills test.
    [Show full text]
  • Zionist Ideology and the Translation of Hebrew Jeffrey M
    Document généré le 2 oct. 2021 22:30 TTR Traduction, terminologie, re?daction Zionist Ideology and the Translation of Hebrew Jeffrey M. Green Idéologie et traduction Résumé de l'article Ideology and Translation Un aspect idéologique de la traduction — La langue d'écriture employée pour Volume 13, numéro 1, 1er semestre 2000 une oeuvre peut avoir une connotation idéologique, susceptible d'être perdue en traduction. Comme c'est le cas lorsqu'il s'agit de langues associées à des URI : https://id.erudit.org/iderudit/037394ar nationalités émergentes et plutôt restreintes, le choix d'écrire en hébreu DOI : https://doi.org/10.7202/037394ar moderne peut être lié à une idéologie nationaliste (le sionisme). Cet article soutient que la création de l'hébreu moderne est semblable à celle d'autres langues minoritaires, qui sont les langues maternelles de communautés Aller au sommaire du numéro relativement peu nombreuses, à la différence des quelques langues très largement répandues et non limitées à un seul pays. Le développement et l'usage d'une langue minoritaire sont l'expression d'une affirmation de soi qui Éditeur(s) entraîne un certain isolement. L'auteur étudie une oeuvre du romancier israélien Aharon Megged et fait état de la signification idéologique de la langue Association canadienne de traductologie d'écriture (l'hébreu moderne) en tant qu'élément littéraire du roman. Il note les aspects de cette signification qui seraient gommés par la traduction et ISSN souligne que les traducteurs doivent être conscients de ce problème. 0835-8443 (imprimé) 1708-2188 (numérique) Découvrir la revue Citer cet article Green, J.
    [Show full text]
  • 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.
    [Show full text]
  • 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.
    [Show full text]
  • 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.
    [Show full text]
  • 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.
    [Show full text]
  • 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.
    [Show full text]
  • 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.
    [Show full text]
  • 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.
    [Show full text]