Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice

[1]  G. Węgrzyn,et al.  Glycosaminoglycans and mucopolysaccharidosis type III. , 2016, Frontiers in bioscience.

[2]  P. Gissen,et al.  Intracerebroventricular Cerliponase Alfa (BMN 190) in Children with CLN2 Disease: Interim Results from a Phase 1/2, Open-Label, Dose-Escalation Study , 2016 .

[3]  W. Pardridge,et al.  Insulin Receptor Antibody-α-N-Acetylglucosaminidase Fusion Protein Penetrates the Primate Blood-Brain Barrier and Reduces Glycosoaminoglycans in Sanfilippo Type B Fibroblasts. , 2016, Molecular pharmaceutics.

[4]  P. Fitzpatrick,et al.  Histologic characterization of the progression of central nervous system pathology in the mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo syndrome type B) mouse model and bio-distribution and efficacy of the intracerebroventricular enzyme replacement therapy, BMN 250 , 2016 .

[5]  L. Lue,et al.  Immune phenotypes of microglia in human neurodegenerative disease: challenges to detecting microglial polarization in human brains , 2015, Alzheimer's Research & Therapy.

[6]  V. Haurigot,et al.  Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy. , 2015, Human molecular genetics.

[7]  P. Trim,et al.  Delivery of therapeutic protein for prevention of neurodegenerative changes: Comparison of different CSF-delivery methods , 2015, Experimental Neurology.

[8]  W. Banks,et al.  Intrathecal delivery of protein therapeutics to the brain: a critical reassessment. , 2014, Pharmacology & therapeutics.

[9]  P. Fitzpatrick,et al.  Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB , 2014, Proceedings of the National Academy of Sciences.

[10]  C. Heldermon,et al.  Mucopolysaccharidosis III (Sanfilippo Syndrome)- disease presentation and experimental therapies. , 2014, Pediatric endocrinology reviews : PER.

[11]  P. Dickson,et al.  Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts. , 2014, The Biochemical journal.

[12]  J. Esko,et al.  Glycan-based biomarkers for mucopolysaccharidoses. , 2014, Molecular genetics and metabolism.

[13]  A. Quinn,et al.  SBC-103, a recombinant human alpha-N-acetylglucosaminidase, demonstrates mannose-6-phosphate receptor dependent transport in an in vitro blood-brain barrier model , 2014 .

[14]  P. Fitzpatrick,et al.  Engineering of a recombinant NAGLU fusion protein with insulin-like growth factor 2 leads to improved cellular uptake via a glycosylation-independent lysosomal targeting pathway , 2014 .

[15]  John L. Orrock,et al.  Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice , 2013, Gene Therapy.

[16]  B. Byrne,et al.  Glycosylation-independent Lysosomal Targeting of Acid α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice* , 2012, The Journal of Biological Chemistry.

[17]  Kevin W Eliceiri,et al.  NIH Image to ImageJ: 25 years of image analysis , 2012, Nature Methods.

[18]  J. Cooper,et al.  Neuropathology in Mouse Models of Mucopolysaccharidosis Type I, IIIA and IIIB , 2012, PloS one.

[19]  S. Fumagalli,et al.  Temporal pattern of expression and colocalization of microglia/macrophage phenotype markers following brain ischemic injury in mice , 2011, Journal of Neuroinflammation.

[20]  E. Neufeld,et al.  Defects in the Medial Entorhinal Cortex and Dentate Gyrus in the Mouse Model of Sanfilippo Syndrome Type B , 2011, PloS one.

[21]  J. Esko,et al.  Disease-Specific Non-Reducing End Carbohydrate Biomarkers for Mucopolysaccharidoses , 2011, Nature chemical biology.

[22]  F. Wijburg,et al.  Mucopolysaccharidosis type III (Sanfilippo Syndrome): emerging treatment strategies. , 2011, Current Pharmaceutical Biotechnology.

[23]  H. Fu,et al.  Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery. , 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

[24]  S. Raoul,et al.  Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes. , 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

[25]  Carmela Di Domenico,et al.  Serum MIP-1 α level: a biomarker for the follow-up of lentiviral therapy in mucopolysaccharidosis IIIB mice , 2010, Journal of Inherited Metabolic Disease.

[26]  L. Kudo,et al.  Sanfilippo syndrome type B, a lysosomal storage disease, is also a tauopathy , 2009, Proceedings of the National Academy of Sciences.

[27]  Frederico A. C. Azevedo,et al.  Equal numbers of neuronal and nonneuronal cells make the human brain an isometrically scaled‐up primate brain , 2009, The Journal of comparative neurology.

[28]  J. Hopwood,et al.  Examination of intravenous and intra‐CSF protein delivery for treatment of neurological disease , 2009, The European journal of neuroscience.

[29]  Roger Lawrence,et al.  Disaccharide structure code for the easy representation of constituent oligosaccharides from glycosaminoglycans , 2008, Nature Methods.

[30]  B. Poorthuis,et al.  Sanfilippo syndrome: A mini-review , 2008, Journal of Inherited Metabolic Disease.

[31]  D. Dickson,et al.  Actin-binding Proteins Coronin-1a and IBA-1 are Effective Microglial Markers for Immunohistochemistry , 2007, The journal of histochemistry and cytochemistry : official journal of the Histochemistry Society.

[32]  M. Mcentee,et al.  Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid. , 2007, Molecular genetics and metabolism.

[33]  S. Ryazantsev,et al.  Lysosomal accumulation of SCMAS (subunit c of mitochondrial ATP synthase) in neurons of the mouse model of mucopolysaccharidosis III B. , 2007, Molecular genetics and metabolism.

[34]  A. Cressant,et al.  Improved Behavior and Neuropathology in the Mouse Model of Sanfilippo Type IIIB Disease after Adeno-Associated Virus-Mediated Gene Transfer in the Striatum , 2004, The Journal of Neuroscience.

[35]  W. Mobley,et al.  Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I. , 2004, Molecular genetics and metabolism.

[36]  W. Sly,et al.  Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice. , 2004, Proceedings of the National Academy of Sciences of the United States of America.

[37]  S. Kar,et al.  Insulin‐like growth factor‐II/mannose‐6‐phosphate receptor: Widespread distribution in neurons of the central nervous system including those expressing cholinergic phenotype , 2003, The Journal of comparative neurology.

[38]  S. Ryazantsev,et al.  Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB , 2003, Proceedings of the National Academy of Sciences of the United States of America.

[39]  M. Fanselow,et al.  Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N-acetylglucosaminidase. , 1999, Proceedings of the National Academy of Sciences of the United States of America.

[40]  M. Alexander,et al.  Principles of Neural Science , 1981 .

[41]  A. Fensom,et al.  4‐methylumbelliferyl α‐N‐acetylglucosaminidase activity for diagnosis of Sanfilippo B disease , 1985 .

[42]  K. Vonfigura Human alpha-n-acetylglucosaminidase. 2. Activity towards natural substrates and multiple recognition forms. , 1977 .

[43]  R. Giugliani,et al.  The mucopolysaccharidoses. , 1976, Journal of medical genetics.

[44]  R. Crystal,et al.  Intracerebral administration of rAAV2/5hNAGLU vector in children with MPS IIIB: results at 30 months of a phase I/II trial , 2017 .

[45]  J. Hopwood,et al.  Delivery of recombinant proteins via the cerebrospinal fluid as a therapy option for neurodegenerative lysosomal storage diseases. , 2009, International journal of clinical pharmacology and therapeutics.

[46]  A. Fensom,et al.  4-Methylumbelliferyl alpha-N-acetylglucosaminidase activity for diagnosis of Sanfilippo B disease. , 1985, Clinical genetics.

[47]  K. von Figura Human alpha-n-acetylglucosaminidase. 2. Activity towards natural substrates and multiple recognition forms. , 1977, European journal of biochemistry.

[48]  Anthony O. Fedele The Application of Clinical Genetics Dovepress Sanfilippo Syndrome: Causes, Consequences, and Treatments , 2022 .