Gene Therapy of the Central Nervous System

From Bench to Bedside

Publisher: Academic Press

Written in English
Cover of: Gene Therapy of the Central Nervous System |
Published: Pages: 370 Downloads: 103
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Edition Notes

ContributionsMichael G. Kaplitt (Editor), Matthew During (Editor)
The Physical Object
Number of Pages370
ID Numbers
Open LibraryOL7327687M
ISBN 100123976324
ISBN 109780123976321

Gene Therapy for Misfolding Protein Diseases of the Central Nervous System 23 May | Neurotherapeutics, Vol. 10, No. 3 Direct and Retrograde Transduction of Nigral Neurons with AAV6, 8, and 9 and Intraneuronal Persistence of Viral ParticlesCited by:   A new technique using tiny bubbles and ultrasound waves could ease the way for gene therapy to be delivered to the central nervous system, a mouse study suggests.. The technique had a neuroprotective effect on mice, and those treated to model Parkinson’s showed improvements in measurements of motor function, the study found/5(6). New Advanced Strategies for the Treatment of Lysosomal Diseases Affecting the Central Nervous System Current Pharmaceutical Design, Vol. 25, No. 17 Cell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNSCited by: 8. Thoroughly revised to reflect the latest advances in neurosurgery, radiation oncology, chemotherapy, biological therapy, and the basic sciences, the Second Edition of this highly acclaimed volume is the most comprehensive, current reference on tumors of the central and peripheral nervous system. More than of the foremost authorities present multimodality treatment strategies for specific.

  Routing gene therapy directly into the brain. (hematopoietic) stem cells directly into the brain could herald a revolution in our approach to treating central nervous system diseases and neurodegenerative disorders. director of the gene therapy program at Dana-Farber/Boston Children’s and the senior author of the new study. Advances in gene therapy technology allow for the efficient and widespread delivery of gene therapies to the central nervous system. Prevail is developing adeno-associated virus (AAV)-based gene therapies for the treatment of neurodegenerative diseases. AAVs are small, non-replicating viruses that are not known to cause disease in humans. function. Central nervous system (CNS)-directed gene therapy via gene replacement represents a powerful mo-dality to achieve long-term correction of disorders follow-ing a single treatment. Multiple vectors exist that can be used for gene therapy, including integrating lentiviral vec-tors and non-integrating adeno-associated virus (AAV) vectors Cited by: Gene therapy is one of the most promising approaches for the treatment of lysosomal storage disorders (LSDs). This is especially true for the 75 % of LSDs that have central nervous system (CNS) involvement, where enzyme replacement therapy (ERT), the standard of care for LSDs, is ineffective in treating the neurological features of these diseases.

  The experimental gene therapy is targeting an inherited disorder that afflicts infants and progressively destroys nerve cells in the brain and spinal cord progressive central nervous system.   Gene therapy protects against age-related cognitive, memory deficits to be safe and effective in the implementation of a central nervous system gene therapy. In fact, the Food and Drug.

Gene Therapy of the Central Nervous System Download PDF EPUB FB2

This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials.

Gene Therapy of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this 5/5(2).

Gene Therapy of the Central Nervous System: From Bench to Bedside - Kindle edition by Michael G. Kaplitt, Matthew During. Download it once and read it on your Kindle device, PC, phones or tablets. Use features like bookmarks, note taking and highlighting while reading Gene Therapy of the Central Nervous System: From Bench to Bedside.5/5(1).

Gene Therapy of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject.

Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and are expanding the promise of gene therapy for the future of basic and clinical neuroscience. The development of gene transfer techniques to the central nervous system (CNS) for therapeutic treatment of a variety of debilitating diseases has greatly diversified in the past dozen years and continues to increase with remarkable momentum.

BOOK TOOLS. Save to My Profile; Recommend to Your Librarian; BOOK MENU. Book Home; ABOUT THIS BOOK. Editors & Contributors; SPECIAL FEATURES. Table of Contents; Standard Article. Gene therapy in the central nervous system. Part 1.

Genetics. Gene Therapy. Specialist Review. Gene Therapy for Central Nervous System in Duchenne Muscular Dystrophy. Authors; Authors and affiliations; Kunkel LM () Localization of dystrophin to postsynaptic regions of central nervous system cortical neurons. Nature ()– Vaillend C., Zarrouki F., Vacca O.

() Gene Therapy for Central Nervous System in Cited by: 1. DELIVERY OF GENE-TARGETED THERAPIES TO THE CENTRAL NERVOUS SYSTEM Sarah DeVos, project leader at Denali Therapeutics, described three different modalities for delivery of gene-targeting therapeutics to the CNS: Naked gene therapy, where the genetic material is delivered without a viral capsid to modulate where the gene is expressed.

Gene therapy. The central nervous system (CNS) is a complex organ with discrete and intricate interconnections between various types of neurons, glia, and other cells. At first sight, CNS seems to be a rather complicated target for gene therapeutic by: 5.

Genetic therapy covers a range of methods for modifying the nervous system, including delivery of genes, sequence-targeted regulatory molecules, genetically modified cells and oligonucleotides.

In this review, we focus on ways to change the genetic status of the nervous system, including routes of access and modes of by:   Optimizing promoters for rAAV-mediated gene expression in the peripheral and central nervous system using self-complementary vectors.

Hum Gene Ther ; – CASCited by:   Gene Therapy in the Central Nervous System: The Use of Recombinant Retroviruses. Steven T. Suhr, PhD; Fred H. Gage, PhD. Author Affiliations Article Information. From the Laboratory of Genetics, Salk Institute for Biological Studies, La Jolla, Calif.

Cited by: Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII Brittney L Gurda1, Adrien De Guilhem De Lataillade2, Peter Bell 3, Yanqing Zhu, Hongwei Yu, Ping Wang 1, Jessica Bagel, Charles H Vite, Tracey Sikora1,4, Christian Hinderer 3, Roberto Calcedo.

24 Gene Therapy for Cent ral Nervous System in Duchenne Muscula r Dystrophy sites and/or the use of distinct delivery routes, different AA V serotypes, or naked. Gene Therapy of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject.

Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and are expanding the promise of gene therapy for the future of basic and clinical : Elsevier Science.

PLANNING COMMITTEE ON ADVANCING GENE THERAPY FOR NERVOUS SYSTEM DISORDERS 1. STORY LANDIS (Co-Chair), Director Emeritus, National Institute of Neurological Disorders and Stroke.

LAMYA SHIHABUDDIN (Co-Chair), Sanofi. ZESHAN AHMED, Eli Lilly and Company. DAVID BREDT, Janssen Research & Development, LLC. DANIEL BURCH, PPD Biotech. JOSEPH BUXBAUM, Icahn. The Forum on Neuroscience and Nervous System Disorders hosted a day public workshop on Aprilthat brought together experts and key stakeholders from academia, government, industry, and non-profit organizations and explored approaches for advancing the development of gene therapies for nervous system disorders, including approaches using RNA interference and antisense.

Get this from a library. Gene therapy of the central nervous system: from bench to bedside. [Michael G Kaplitt; Matthew J During;]. Gene Therapy for the Nervous System: Challenges and New Strategies. Instead, in this review our goal is to provide the reader with (1) an overview of the barriers to efficient gene delivery to the central nervous system (CNS) and (2) the state of the art in virus and nonviral-based neurotherapeutics.

Cited by: Ultimately, the principal outcome of gene therapy is to functionally restore damaged neuronal and/or axonal connections irrespective of the system it is being introduced in to. This research topic is devoted to work using gene therapy for the both the central and/or peripheral nervous system.

"Gene Therapy of the Central Nervous System: From Bench to Bedside" represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and are expanding the promise of gene therapy for the future of basic and clinical neuroscience.

Gene Therapy Targeting in the Central Nervous System. Author(s): Jorge A. Benitez, Jose by: It is with great pleasure that we present the research topic dedicated to Gene Therapy for the Central (CNS) and Peripheral Nervous System (PNS).

Gene therapy is at the cutting-edge of techniques utilised to develop novel therapeutics to treat insult(s) to the brain, spinal cord and/or peripheral nerves. Indeed, gene therapy can be applied via many different routes and as such can overcome the Cited by: 1.

Genetic Manipulation of the Nervous System: An Overview. Non-Neurotropic Adenovirus: A Vector for the Gene Transfer to the Brain and Possible Gene Therapy of Neurological Disorders.

Adenovirus: A New Tool to Transfer Genes Into the Central Nervous System for Treatment of. Nervous System and Muscle Gene Therapy Sourav R Choudhury 1,2, Zachary Fitzpatrick 3, Anne F Harris, Stacy A Maitland, Jennifer S Ferreira, Yuanfan Zhang 4, Shan Ma 2,5, Rohit B Sharma 6, Heather L Gray-Edwards 7, Jacob A Johnson 8.

The infection characteristic for the nervous system makes adenovirus a useful gene therapy tool for neurological disorders. 44 Many studies have revealed that adenovirus can be maintained in the.

It is with great pleasure that we present the research topic dedicated to Gene Therapy for the Central (CNS) and Peripheral Nervous System (PNS).

Gene therapy is at the cutting-edge of techniques utilized to develop novel therapeutics to treat insult(s) to the brain, spinal cord and/or peripheral by: 1.

Routing gene therapy directly into brain Researchers report that transplanting genetically-engineered cells directly into the brain could help treat a variety of central nervous system. If you have advanced Parkinson’s disease and are experiencing dyskinesia symptoms or motor fluctuations that are not responding to medicines, you may be eligible for a clinical trial examining a new minimally invasive method to treat your symptoms.

Next Generation Strategies for Gene-Targeted Therapies of Central Nervous System Disorders. Please book early because space is limited. The rate will be available until Friday, Aug or until Gene Therapy" group rate.

Dining: Please note that no meals will be provided at the meeting. Many dining options are available within. Sandhoff disease (SD) is caused by deficiency of N-acetyl-β-hexosaminidase (Hex) resulting in pathological accumulation of GM2 ganglioside in lysosomes of the central nervous system Cited by:.

Application of gene therapies is a promising approach to the treatment of various neurological disorders, including Parkinson's disease, amyotrophic lateral sclerosis (ALS), and lysosomal storage disorders, which are not treatable by any other means.

However, the blood–brain barrier (BBB) is a key obstacle to gene delivery to the central nervous system (CNS).Cited by: 1. Passage Bio reeled in a $ million series A on Thursday, which will support the development of five programs aimed at treating rare monogenic central nervous system (CNS) diseases.

Passage Bio. Recent advances have increased our molecular understanding of the central nervous system (CNS), in both health and disease.

In order to realize the clinical benefits of these findings, new molecular‐based therapies need to be developed, such as CNS gene by: