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Journal Article
PLoS ONE, ISSN 1932-6203, 11/2014, Volume 9, Issue 11, p. e114809
Journal Article
2004, ISBN 0198508786, xxvii. 447
Lysosomal storage diseases are inherited metabolic disorders characterized by severe pathology, typically involving the brain. Although individually rare, they... 
Brain | Lysosomal storage diseases | Treatment | Metabolism | Disorders | neuroscience
Book
The Journal of Cell Biology, ISSN 0021-9525, 4/2004, Volume 165, Issue 1, pp. 123 - 133
Journal Article
Materials Today, ISSN 1369-7021, 10/2012, Volume 15, Issue 10, pp. 436 - 442
In order to elicit therapeutic effects, many drugs including small molecule anticancer drugs, proteins, siRNA, and DNA have to be delivered and released into... 
TRIGGERED RELEASE | BIODEGRADABLE MICELLES | DIBLOCK COPOLYMER | MATERIALS SCIENCE, MULTIDISCIPLINARY | LYSOSOMAL-ENZYMES | RESPONSIVE NANOPARTICLES | NONVIRAL GENE TRANSFECTION | CROSS-LINKED MICELLES | BLOCK-COPOLYMER | CANCER-THERAPY | PROTEIN DELIVERY | Drugs | Care and treatment | Cells | Cancer
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2017, Volume 25, Issue 5, pp. 1155 - 1162
Lysosomal storage disorders (LSDs) are a broad class of monogenic diseases with an overall incidence of 1:7,000 newborns, due to the defective activity of one... 
gene therapy | lysosomal enzymes | storage diseases | hematopoietic stem cells | lentiviral vectors | central nervous system | MEDICINE, RESEARCH & EXPERIMENTAL | HURLER-SYNDROME | BONE-MARROW-TRANSPLANTATION | METACHROMATIC LEUKODYSTROPHY | MUCOPOLYSACCHARIDOSIS TYPE-II | X-LINKED ADRENOLEUKODYSTROPHY | BLOOD-BRAIN-BARRIER | ENZYME-REPLACEMENT-THERAPY | LYSOSOMAL-ENZYME | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | MOUSE MODEL | GENETICS & HEREDITY | A-DEFICIENT MICE | Hydrolases - genetics | Lysosomal Storage Diseases - enzymology | Humans | Brain Damage, Chronic - therapy | Brain Damage, Chronic - enzymology | Brain Damage, Chronic - genetics | Transplantation, Homologous | Lentivirus - metabolism | Lysosomes - metabolism | Lentivirus - genetics | Brain Damage, Chronic - pathology | Disease Models, Animal | Gene Expression | Lysosomal Storage Diseases - therapy | Genetic Vectors - chemistry | Genetic Vectors - metabolism | Hematopoietic Stem Cell Transplantation | Lysosomal Storage Diseases - genetics | Hematopoietic Stem Cells - metabolism | Hydrolases - deficiency | Lysosomal Storage Diseases - pathology | Enzyme Replacement Therapy - methods | Animals | Hematopoietic Stem Cells - cytology | Genetic Therapy - methods | Neonates | Brain | Enzymes | Disease | Clinical trials | Lysosomes | Lysosomal storage diseases | Lysergide | Medical screening | Patients | Hematopoietic stem cells | Hemopoiesis | Defects | Storage diseases | Proteins | Genotype & phenotype | Medical prognosis | Stem cells | Fibroblasts | Mutation | Gene therapy | Brain injury | Review
Journal Article
Journal of Pediatric Health Care, ISSN 0891-5245, 05/2018, Volume 32, Issue 3, pp. 285 - 294
Lysosomal storage disorders (LSDs) are a heterogeneous group of approximately 50 rare inherited metabolic conditions that result from enzyme deficiencies that... 
lysosomal enzyme disorder | lysosomal storage disease | newborn screening | Lysosomal storage disorder | lysosomal enzyme disorder | MUCOPOLYSACCHARIDOSES | NURSING | PEDIATRICS | HEALTH POLICY & SERVICES | KRABBE-DISEASE | Infants (Newborn) | Enzymes
Journal Article
NATURE COMMUNICATIONS, ISSN 2041-1723, 09/2019, Volume 10, Issue 1, pp. 4045 - 14
Journal Article
Pediatric Research, ISSN 0031-3998, 07/2019, Volume 86, Issue 1, pp. 85 - 91
BACKGROUND: I-cell disease is characterized by the presence of vacuole-like inclusions in lymphocytes. However, the nature and clinical significance of these... 
DIAGNOSIS | PSEUDO-HURLER POLYDYSTROPHY | DEFICIENT | LYSOSOMAL-ENZYMES | PEDIATRICS | MUTATIONS | FIBROBLASTS | LYMPHOCYTES
Journal Article