Download or read book Enzyme Therapy in Lysosomal Storage Diseases written by Joseph Michel Tager and published by . This book was released on 1974 with total page 332 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Enzyme Therapy in Lysosomal Storage Diseases written by J. M. Tager and published by . This book was released on 1974 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Enzyme Therapy in Lysosomal Storage Diseases Proceedings of the Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases Leiden The Netherlands April 2 3 1974 written by J. M. Tager and published by . This book was released on 1974 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Enzyme therapy in lysosomal storage diseases proceedings for the Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases Leiden written by Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases, Leyden, 1974 and published by . This book was released on with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Proceedings of the Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases Leiden The Netherlands April 2 3 1974 and Papers Presented at a Boerhaave Advanced Course on Lysosomal Enzymopathies Leiden The Netherlands April 4 5 1974 written by W. Th Daems and published by . This book was released on 1974 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Enzyme Therapy in Lysosomal Storage Diseases written by Joseph Michael Tager and published by . This book was released on 1974 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Current Catalog written by National Library of Medicine (U.S.) and published by . This book was released on with total page 1430 pages. Available in PDF, EPUB and Kindle. Book excerpt: First multi-year cumulation covers six years: 1965-70.
Download or read book Enzyme Therapy in Lysosomal Storage Diseases written by and published by . This book was released on 1974 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book National Library of Medicine Current Catalog written by National Library of Medicine (U.S.) and published by . This book was released on 1971 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt: First multi-year cumulation covers six years: 1965-70.
Download or read book Lysosomal Storage Disorders written by John A. Barranger and published by Springer Science & Business Media. This book was released on 2007-10-16 with total page 563 pages. Available in PDF, EPUB and Kindle. Book excerpt: The knowledge of lysosomal biology and the consequences of its dysfunction have increased dramatically in the past 60 years. This book describes the nature of the lysosomal dysfunction and diseases as well as potential future treatments and therapies. Disease specific chapters provide thorough reviews of the clinical features of lysosomal storage disorders, their molecular basis and the commercial or experimental therapeutic approaches sought in this area. This is an invaluable resource for researchers in biochemical and molecular genetics, enzyme therapy, and gene transfer.
Download or read book Lysosomal Storage Disorders written by Atul B. Mehta and published by John Wiley & Sons. This book was released on 2012-09-06 with total page 1 pages. Available in PDF, EPUB and Kindle. Book excerpt: The last two decades have seen a huge expansion in research in the area of lysosomal storage disorders, which has substantially extended our understanding of both the scientific and the clinical basis of these diseases. Lysosomal Storage Disorders: A Practical Guide is the fruit of an ambitious project aiming to review both the scientific and the clinical aspects of lysosomal storage disorders, resulting in this accessible volume, which gives an up-to-date overview of the subject. There is substantial scientific interest in these diseases: new advances in small molecule therapy are likely to be useful in the near future, and trials are already underway. Lysosomal storage disorders offer a unique platform for teaching modern clinical science, from basic genetics through to clinical applications. The first part of the book reviews and classifies our current understanding of the physiology and pathophysiology of lysosomal storage disorders. The second part of the book reviews individual diseases, and gives perspectives from patients and experts looking towards future therapeutic directions. Lysosomal Storage Disorders: A Practical Guide is the ideal guide for a wide audience including scientists, clinicians, health care workers and administrators, those working in the pharmaceutical industry, patients and their organisations. Titles of related interest Haematology at a Glance • Mehta • ISBN 9781405179706 Atlas of Endocrine and Metabolic Disease • Pozzilli • ISBN 9780470656273
Download or read book Enzyme Replacement Therapies for Lysosomal Storage Diseases written by U. S. Department of Health and Human Services and published by Createspace Independent Pub. This book was released on 2013-03-24 with total page 112 pages. Available in PDF, EPUB and Kindle. Book excerpt: Lysosomes are generally spherical, subcellular organelles bounded by a single layer membrane within eukaryotic cells. They are ubiquitous structures that contain an array of glycoprotein acid hydrolase enzymes, all of which are synthesized in the endoplasmic reticulum and modified in the Golgi apparatus. Lysosomal enzymes catabolize all major classes of biological macromolecules such as proteins, nucleic acids, glycosphingolipids, mucopolysaccharides, and glycogen, as well as sequestered bacteria, viruses, and other foreign substances that are taken up by phagocytosis into white blood cells and macrophages. Lysosomes are also responsible for autophagy, the gradual turnover of each cell's own components as they age and become obsolescent. They may be considered the main site of intracellular digestion and housekeeping. Lysosomal storage diseases (LSDs) comprise a group of unique monogenic autosomal or Xlinked diseases that occur secondary to genetic defects (e.g., single nucleotide substitutions, frameshift mutations, gene deletions) that cause total deficiency or reduced activity of specific native enzymes within the lysosomes. This allows macromolecular compounds that are normally enzymatically catabolized to accumulate within these organelles, expanding them and causing progressive damage in connective tissue, skeletal structure, various organs, and, in some cases, the central nervous system. The damage caused by substrate accumulation results in physical deterioration, functional impairment, and, potentially, death. Some fifty different LSDs have been identified, broadly divided into categories that are defined by accumulation of a specific macromolecule. Although each LSD is individually somewhat rare, as a group they have an incidence of about 1 per 7,000 to 8,000 live births, with regional and genetic population variations. LSDs may be variably expressed as infantile, juvenile, or adult forms. In adult-onset diseases, the pathogenesis is usually slower than in the infantile or juvenile forms, and may include peripheral and CNS symptoms. By contrast, infantile and juvenile forms often involve progressive central nervous system involvement in addition to peripheral symptoms. LSDs also often exhibit significant heterogeneity in ultimate expression, with early or late presentation of symptomatic pathology that may be a function of mutation type and residual enzyme levels. Although specific mutations or types of mutations may be connected to discrete disease effects, genotype-phenotype correlations are often not strong. The objective of this Technical Brief is to provide an overview of FDA-approved ERT for the treatment of six lysosomal storage diseases (shown in Table 1). Four Guiding Questions (following) address the clinical indications for each ERT, potential benefits and harms associated with each ERT product, and dosing and administration details of each ERT. An electronic scan of the literature provides a picture of published evidence on clinical use of these agents for each LSD. This Technical Brief also discusses unresolved or controversial issues surrounding the use of ERT to treat lysosomal storage diseases, based on the literature and information obtained through semi-structured, one-on-one telephone interviews with Key Informants. Guiding Questions include: 1. What FDA-approved enzyme-replacement therapy (ERT) products are available for lysosomal storage diseases (LSDs)? 2. What is the context in which each FDA-approved ERT product is used? 3. What published and unpublished studies have reported on the use and safety of this intervention? 4. What are key unresolved or controversial issues with ERT in LSDs?
Download or read book Lysosomal Storage Disorders written by John A. Barranger and published by Springer. This book was released on 2007-10-01 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: The knowledge of lysosomal biology and the consequences of its dysfunction have increased dramatically in the past 60 years. This book describes the nature of the lysosomal dysfunction and diseases as well as potential future treatments and therapies. Disease specific chapters provide thorough reviews of the clinical features of lysosomal storage disorders, their molecular basis and the commercial or experimental therapeutic approaches sought in this area. This is an invaluable resource for researchers in biochemical and molecular genetics, enzyme therapy, and gene transfer.
Download or read book Molecular Basis of Lysosomal Storage Disorders written by John Barranger and published by Elsevier. This book was released on 2012-12-02 with total page 523 pages. Available in PDF, EPUB and Kindle. Book excerpt: Molecular Basis of Lysosomal Storage Disorders contains the proceedings of the 1983 Conference on the Molecular Basis of Lysosomal Storage Disorders, held at the National Institutes of Health in Bethesda, Maryland. The papers focus on the molecular biology of, and therapeutic approaches to, lysosomal storage disorders, such as mucopolysaccharidoses, sphingolipidoses, and Gaucher disease. Organized into six sections comprised of 29 chapters, this book begins with an overview of enzymes, activator proteins, and stabilizers that underlie lysosomal storage disorders. It then discusses some developments in enzyme purification, receptors for glycoprotein enzymes, factors that control endocytosis, and the intracellular fate of lysosomal hydrolases. Some chapters explain the enzyme biosynthesis, bone marrow transplantation, and enzyme replacement, along with cell hybridization, chromosome localization, phenotype discrimination, and cloning of genes for human lysosomal enzymes. This book is helpful to biochemists, physiologists, pathologists, geneticists, clinical investigators, and practicing physicians concerned with the study, care, and treatment of patients with hereditary metabolic disorders, as well as undergraduate and graduate level students involved in research in this discipline.
Download or read book Nanotechnology in Drug Delivery written by Melgardt M. de Villiers and published by Springer Science & Business Media. This book was released on 2008-10-29 with total page 681 pages. Available in PDF, EPUB and Kindle. Book excerpt: The reader will be introduced to various aspects of the fundamentals of nanotechnology based drug delivery systems and the application of these systems for the delivery of small molecules, proteins, peptides, oligonucleotides and genes. How these systems overcome challenges offered by biological barriers to drug absorption and drug targeting will also be described.
Download or read book Lysosomal Storage Diseases Advances in Research and Treatment 2011 Edition written by and published by ScholarlyEditions. This book was released on 2012-01-09 with total page 17 pages. Available in PDF, EPUB and Kindle. Book excerpt: Lysosomal Storage Diseases: Advances in Research and Treatment: 2011 Edition is a ScholarlyPaper™ that delivers timely, authoritative, and intensively focused information about Lysosomal Storage Diseases in a compact format. The editors have built Lysosomal Storage Diseases: Advances in Research and Treatment: 2011 Edition on the vast information databases of ScholarlyNews.™ You can expect the information about Lysosomal Storage Diseases in this eBook to be deeper than what you can access anywhere else, as well as consistently reliable, authoritative, informed, and relevant. The content of Lysosomal Storage Diseases: Advances in Research and Treatment: 2011 Edition has been produced by the world’s leading scientists, engineers, analysts, research institutions, and companies. All of the content is from peer-reviewed sources, and all of it is written, assembled, and edited by the editors at ScholarlyEditions™ and available exclusively from us. You now have a source you can cite with authority, confidence, and credibility. More information is available at http://www.ScholarlyEditions.com/.
Download or read book National Union Catalog written by and published by . This book was released on with total page 528 pages. Available in PDF, EPUB and Kindle. Book excerpt: Includes entries for maps and atlases.
