Literature DB >> 24584077

A Hitchhiker's guide to the blood-brain barrier: in trans delivery of a therapeutic enzyme.

Mark S Sands1.   

Abstract

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Year:  2014        PMID： 24584077      PMCID： PMC3944337          DOI： 10.1038/mt.2014.12

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  17 in total

1.  Abnormal immune function in vivo in a murine model of lysosomal storage disease.

Authors:  T M Daly; R G Lorenz; M S Sands
Journal:  Pediatr Res       Date:  2000-06       Impact factor: 3.756

2.  Impact of high-dose, chemically modified sulfamidase on pathology in a murine model of MPS IIIA.

Authors:  Tina Rozaklis; Helen Beard; Sofia Hassiotis; Antony R Garcia; Matthew Tonini; Amanda Luck; Jing Pan; Justin C Lamsa; John J Hopwood; Kim M Hemsley
Journal:  Exp Neurol       Date:  2011-04-16       Impact factor: 5.330

3.  Association of mutations in a lysosomal protein with classical late-infantile neuronal ceroid lipofuscinosis.

Authors:  D E Sleat; R J Donnelly; H Lackland; C G Liu; I Sohar; R K Pullarkat; P Lobel
Journal:  Science       Date:  1997-09-19       Impact factor: 47.728

4.  Inborn errors of mucopolysaccharide metabolism.

Authors:  E F Neufeld; J C Fratantoni
Journal:  Science       Date:  1970-07-10       Impact factor: 47.728

Review 5.  The phosphomannosyl recognition system for intracellular and intercellular transport of lysosomal enzymes.

Authors:  W S Sly; H D Fischer
Journal:  J Cell Biochem       Date:  1982       Impact factor: 4.429

6.  Human beta-glucuronidase: in vivo clearance and in vitro uptake by a glycoprotein recognition system on reticuloendothelial cells.

Authors:  D T Achord; F E Brot; C E Bell; W S Sly
Journal:  Cell       Date:  1978-09       Impact factor: 41.582

7.  Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

Authors:  Carole Vogler; Beth Levy; Jeffrey H Grubb; Nancy Galvin; Yun Tan; Emil Kakkis; Nadine Pavloff; William S Sly
Journal:  Proc Natl Acad Sci U S A       Date:  2005-09-14       Impact factor: 11.205

8.  Enzyme replacement therapy for murine mucopolysaccharidosis type VII.

Authors:  M S Sands; C Vogler; J W Kyle; J H Grubb; B Levy; N Galvin; W S Sly; E H Birkenmeier
Journal:  J Clin Invest       Date:  1994-06       Impact factor: 14.808

9.  Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier.

Authors:  Akihiko Urayama; Jeffrey H Grubb; William S Sly; William A Banks
Journal:  Proc Natl Acad Sci U S A       Date:  2004-08-16       Impact factor: 11.205

10.  Chemically modified beta-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII.

Authors:  Jeffrey H Grubb; Carole Vogler; Beth Levy; Nancy Galvin; Yun Tan; William S Sly
Journal:  Proc Natl Acad Sci U S A       Date:  2008-02-11       Impact factor: 11.205
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  7 in total

1.  Guanidinylated Neomycin Conjugation Enhances Intranasal Enzyme Replacement in the Brain.

Authors:  Wenyong Tong; Chrissa A Dwyer; Bryan E Thacker; Charles A Glass; Jillian R Brown; Kristina Hamill; Kelley W Moremen; Stéphane Sarrazin; Philip L S M Gordts; Lara E Dozier; Gentry N Patrick; Yitzhak Tor; Jeffrey D Esko
Journal:  Mol Ther       Date:  2017-08-12       Impact factor: 11.454

2.  Engineered antibody therapies coming of age for aging brains.

Authors:  Anne Messer
Journal:  Mol Ther       Date:  2014-10       Impact factor: 11.454

3.  Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity.

Authors:  John Marshall; Jennifer B Nietupski; Hyejung Park; James Cao; Dinesh S Bangari; Cristina Silvescu; Terry Wilper; Kristen Randall; Drew Tietz; Bing Wang; Xiaoyou Ying; John P Leonard; Seng H Cheng
Journal:  Mol Ther       Date:  2019-06-04       Impact factor: 11.454

Review 4.  Recent advances in gene therapy for lysosomal storage disorders.

Authors:  David Pw Rastall; Andrea Amalfitano
Journal:  Appl Clin Genet       Date:  2015-06-24

5.  Peptide carrier-mediated non-covalent delivery of unmodified cisplatin, methotrexate and other agents via intravenous route to the brain.

Authors:  Gobinda Sarkar; Geoffry L Curran; Jann N Sarkaria; Val J Lowe; Robert B Jenkins
Journal:  PLoS One       Date:  2014-05-21       Impact factor: 3.240

Review 6.  New therapeutic approaches for Krabbe disease: The potential of pharmacological chaperones.

Authors:  Samantha J Spratley; Janet E Deane
Journal:  J Neurosci Res       Date:  2016-11       Impact factor: 4.164

7.  Cerebral Spinal Fluid levels of Cytokines are elevated in Patients with Metachromatic Leukodystrophy.

Authors:  Kathryn A Thibert; Gerald V Raymond; Jakub Tolar; Weston P Miller; Paul J Orchard; Troy C Lund
Journal:  Sci Rep       Date:  2016-04-15       Impact factor: 4.379
  7 in total

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