6 edition of Gene therapy in the central nervous system found in the catalog.
Gene therapy in the central nervous system
|Statement||edited by Michael G. Kaplitt and Matthew J. During.|
|The Physical Object|
|Pagination||xiv, 355 p. :|
|Number of Pages||355|
Primary central nervous system lymphoma (PCNSL) is an uncommon non‐Hodgkin lymphoma for which multiple modalities of treatment are often used, including radiation therapy, chemotherapy with or without autologous stem cell transplant (ASCT), and immunotherapy. Neural stem cells – a versatile tool for cell replacement and gene therapy in the central nervous system. V Ourednik. Department of Neurology (Division of Neuroscience), Harvard Medical School, Children's Hospital, Boston, MA, USA, Search for more papers by this author. J Ourednik.
Getting gene therapy to the brain Using a large animal model of genetic brain disease, researchers led by John H. Wolfe of the School of Veterinary Medicine, Perelman School of Medicine, and Children’s Hospital of Philadelphia delivered an effective treatment across the blood-brain barrier to . No therapy for clinical applications as yet. Injuries or diseases of nerves in the central nervous system result in lifelong disabilities, such as paraplegia caused .
The novel gene therapy works by converting astrocytes in the CNS into functioning neurons. Astrocytes are a type of glial cells that are present . Samaranch L, Salegio EA, San Sebastian W, et al. Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Hum Gene Ther ;– Link, Google Scholar; 8. Kells AP, Hadaczek P, Yin D, et al. Efficient gene therapy-based method for the delivery of therapeutics to primate cortex.
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Gene therapy using viral vectors targeting primary sensory neurons or the central nervous system is fast becoming a promising therapeutic option.
In vivo pre-clinical studies show that various vectors reliably infect pain related neurons and gene expression is observed for many weeks. Several groups have already demonstrated the feasibility of viral vector mediated delivery of enkephalin, mu-opioid.
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.5/5(2).
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 by: 1.
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.
Abstract: Targeted gene therapy aims at achieving the expression of therapeutic transgenes in specific and restricted cell populations, thus sparing all other cells of the unwanted effects of the gene product. This strategy is particularly appealing for therapy of the central nervous system (CNS), where many different cell types exist, and where the inappropriate expression of a molecule can Cited by: It includes descriptions of lentiviral and AAV vector development, of therapeutic gene selection (including siRNAs, shRNAs, and microRNAs), and of the most recent clinical applications of gene therapy for diseases of the eye, the cardiovascular system, and the central nervous system.
This characteristic, and extended high-level gene expression, makes them ideal therapeutic agents for the transfer of novel genetic material to the adult central nervous system (CNS).
The weakness of HIV-based vectors, however, is that they currently require stringent biosafety procedures to produce safe recombinant virus. 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. Gene therapy and ASOs have the potential to offer new therapeutic opportunities, and possibly cures, to patients where few or no treatment options exist for their disease.
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.
The primary treatment for MPS II, enzyme replacement therapy, is not effective for central nervous system (CNS) symptoms, such as intellectual disability, because the drugs do not cross the blood–brain barrier.
Recently, autophagy has been associated with LSDs. Gene-Targeted Therapy Approaches for Central Nervous System Disorders: Opportunities and Challenges. Highlights. Gene-targeted therapies include those that target RNA or DNA and either a gene itself or a modulator of that gene (Davidson).
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 Therapy of the Central Nervous System: From Bench to Bedsiderepresents the first definitive volume on this subject.
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. Gene therapy of the central nervous system. Amsterdam ; Boston: Academic Press, (OCoLC) Online version: Gene therapy of the central nervous system. Amsterdam ; Boston: Academic Press, (OCoLC) Document Type: Book: All Authors / Contributors: Michael G Kaplitt; Matthew J During.
Get this from a library. Gene therapy of the central nervous system: from bench to bedside. [Michael G Kaplitt; Matthew J During;]. On April 23 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC.
The past few years have witnessed rapid developments in viral-mediated gene replacement therapy for pediatric central nervous system neurogenetic disorders. Here, we provide practicing pediatric neurologists with an up-to-date, comprehensive overview of these developments and note emerging trends for future research.
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. 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.
BioSpace Gene therapy pioneer uniQure launched the first-in-human adeno-associated virus-based gene therapy clinical trial for Huntington’s disease.
In June, it dosed its first two patients using a novel therapeutic, AMT, delivered directly to the brain.
Initial readouts are expected in. It’s working on adeno-associated vector (AAV) gene therapies for monogenic diseases—that is, diseases caused by a defect in a single gene—of the central nervous system.Anti-Angiogenesis, Gene Therapy, and Immunotherapy in Malignant Gliomas, Tumors of the Central Nervous System - Primary and Secondary, Lee Roy Morgan, IntechOpen, DOI: / Available from: Paula Province, Alexis Bashinski Shaefer, Benjamin McCullough and Hassan M Fathallah-Shaykh (June 11th ).Preclinical animal studies are essential for refining gene therapy methods and learning more about the most effective ways to deliver genes to the central nervous system.
A Gene Therapy Approach.