Literature DB >> 11983877

Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier.

William S Sly1, Carole Vogler.   

Abstract

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Year:  2002        PMID： 11983877      PMCID： PMC122848          DOI： 10.1073/pnas.102175599

Source DB:  PubMed          Journal:  Proc Natl Acad Sci U S A        ISSN： 0027-8424            Impact factor:   11.205


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  30 in total

1.  Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer.

Authors:  A Bosch; E Perret; N Desmaris; D Trono; J M Heard
Journal:  Hum Gene Ther       Date:  2000-05-20       Impact factor: 5.695

2.  Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors.

Authors:  A Bosch; E Perret; N Desmaris; J M Heard
Journal:  Mol Ther       Date:  2000-01       Impact factor: 11.454

3.  Systemic hyperosmolality improves beta-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer.

Authors:  A Ghodsi; C Stein; T Derksen; I Martins; R D Anderson; B L Davidson
Journal:  Exp Neurol       Date:  1999-11       Impact factor: 5.330

4.  A -glucuronidase deficiency mucopolysaccharidosis: studies in cultured fibroblasts.

Authors:  C W Hall; M Cantz; E F Neufeld
Journal:  Arch Biochem Biophys       Date:  1973-03       Impact factor: 4.013

5.  Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis.

Authors:  W S Sly; B A Quinton; W H McAlister; D L Rimoin
Journal:  J Pediatr       Date:  1973-02       Impact factor: 4.406

6.  In vitro correction of deficient human fibroblasts by beta-glucuronidase from different human sources.

Authors:  F E Brot; J H Glaser; K J Roozen; W S Sly; P D Stahl
Journal:  Biochem Biophys Res Commun       Date:  1974-03-15       Impact factor: 3.575

7.  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

8.  Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy.

Authors:  C Vogler; B Levy; N J Galvin; C Thorpe; M S Sands; J E Barker; J Baty; E H Birkenmeier; W S Sly
Journal:  Pediatr Res       Date:  1999-06       Impact factor: 3.756

9.  Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors.

Authors:  Andrew I Brooks; Colleen S Stein; Stephanie M Hughes; Jason Heth; Paul M McCray; Sybille L Sauter; Julie C Johnston; Deborah A Cory-Slechta; Howard J Federoff; Beverly L Davidson
Journal:  Proc Natl Acad Sci U S A       Date:  2002-04-16       Impact factor: 11.205

10.  Phosphohexosyl components of a lysosomal enzyme are recognized by pinocytosis receptors on human fibroblasts.

Authors:  A Kaplan; D T Achord; W S Sly
Journal:  Proc Natl Acad Sci U S A       Date:  1977-05       Impact factor: 11.205
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  13 in total

Review 1.  Endocytic mechanisms for targeted drug delivery.

Authors:  Lisa M Bareford; Peter W Swaan
Journal:  Adv Drug Deliv Rev       Date:  2007-06-28       Impact factor: 15.470

Review 2.  Molecular simulations of carbohydrates and protein-carbohydrate interactions: motivation, issues and prospects.

Authors:  Elisa Fadda; Robert J Woods
Journal:  Drug Discov Today       Date:  2010-06-08       Impact factor: 7.851

Review 3.  Gene therapy for the nervous system: challenges and new strategies.

Authors:  Casey A Maguire; Servio H Ramirez; Steven F Merkel; Miguel Sena-Esteves; Xandra O Breakefield
Journal:  Neurotherapeutics       Date:  2014-10       Impact factor: 7.620

Review 4.  Nanoparticle transport across the blood brain barrier.

Authors:  Andreas M Grabrucker; Barbara Ruozi; Daniela Belletti; Francesca Pederzoli; Flavio Forni; Maria Angela Vandelli; Giovanni Tosi
Journal:  Tissue Barriers       Date:  2016-02-25

5.  Disruption of adaptive energy metabolism and elevated ribosomal p-S6K1 levels contribute to INCL pathogenesis: partial rescue by resveratrol.

Authors:  Hui Wei; Zhongjian Zhang; Arjun Saha; Shiyong Peng; Goutam Chandra; Zenaide Quezado; Anil B Mukherjee
Journal:  Hum Mol Genet       Date:  2010-12-28       Impact factor: 6.150

6.  Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.

Authors:  Baodong Sun; Haoyue Zhang; Daniel K Benjamin; Talmage Brown; Andrew Bird; Sarah P Young; Alison McVie-Wylie; Y-T Chen; Dwight D Koeberl
Journal:  Mol Ther       Date:  2006-09-20       Impact factor: 11.454

Review 7.  Neural stem cell transplantation as a therapeutic approach for treating lysosomal storage diseases.

Authors:  Lamya S Shihabuddin; Seng H Cheng
Journal:  Neurotherapeutics       Date:  2011-10       Impact factor: 7.620

8.  Evaluation of neurodegeneration in a mouse model of infantile batten disease by magnetic resonance imaging and magnetic resonance spectroscopy.

Authors:  Jeeva Munasinghe; Zhongjian Zhang; Eryan Kong; Alison Heffer; Anil B Mukherjee
Journal:  Neurodegener Dis       Date:  2012-02-09       Impact factor: 2.977

9.  Deletion of macrophage-inflammatory protein 1 alpha retards neurodegeneration in Sandhoff disease mice.

Authors:  Yun-Ping Wu; Richard L Proia
Journal:  Proc Natl Acad Sci U S A       Date:  2004-05-20       Impact factor: 11.205

Review 10.  Organelle targeting: third level of drug targeting.

Authors:  Niraj M Sakhrani; Harish Padh
Journal:  Drug Des Devel Ther       Date:  2013-07-17       Impact factor: 4.162
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