Literature DB >> 19361502

Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases.

Shannon L Macauley1, Mark S Sands.   

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Year:  2009        PMID： 19361502      PMCID： PMC2701189          DOI： 10.1016/j.expneurol.2009.03.040

Source DB:  PubMed          Journal:  Exp Neurol        ISSN： 0014-4886            Impact factor:   5.330


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  58 in total

1.  Recognition of human urine alpha-N-acetylglucosaminidase by rat hepatocytes. Involvement of receptors specific for galactose, mannose 6-phosphate and mannose.

Authors:  K Ullrich; R Basner; V Gieselmann; K Von Figura
Journal:  Biochem J       Date:  1979-05-15       Impact factor: 3.857

2.  Isolation of lysosomal alpha-mannosidase mutants of Chinese hamster ovary cells.

Authors:  A R Robbins
Journal:  Proc Natl Acad Sci U S A       Date:  1979-04       Impact factor: 11.205

3.  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

4.  Butanedione treatment reduces receptor binding of a lysosomal enzyme to cells and membranes.

Authors:  L H Rome; J Miller
Journal:  Biochem Biophys Res Commun       Date:  1980-02-12       Impact factor: 3.575

5.  Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease.

Authors:  N W Barton; R O Brady; J M Dambrosia; A M Di Bisceglie; S H Doppelt; S C Hill; H J Mankin; G J Murray; R I Parker; C E Argoff
Journal:  N Engl J Med       Date:  1991-05-23       Impact factor: 91.245

6.  Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy.

Authors:  Darshong Lin; Anthony Donsante; Shannon Macauley; Beth Levy; Carole Vogler; Mark S Sands
Journal:  Mol Ther       Date:  2007-01       Impact factor: 11.454

Review 7.  Niemann-Pick disease.

Authors:  M Elleder
Journal:  Pathol Res Pract       Date:  1989-09       Impact factor: 3.250

8.  Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease.

Authors:  Jean-Pyo Lee; Mylvaganam Jeyakumar; Rodolfo Gonzalez; Hiroto Takahashi; Pei-Jen Lee; Rena C Baek; Dan Clark; Heather Rose; Gerald Fu; Jonathan Clarke; Scott McKercher; Jennifer Meerloo; Franz-Josef Muller; Kook In Park; Terry D Butters; Raymond A Dwek; Philip Schwartz; Gang Tong; David Wenger; Stuart A Lipton; Thomas N Seyfried; Frances M Platt; Evan Y Snyder
Journal:  Nat Med       Date:  2007-03-11       Impact factor: 53.440

9.  Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII.

Authors:  A Donsante; B Levy; C Vogler; M S Sands
Journal:  J Inherit Metab Dis       Date:  2007-02-16       Impact factor: 4.750

10.  Impaired selection of invariant natural killer T cells in diverse mouse models of glycosphingolipid lysosomal storage diseases.

Authors:  Stephan D Gadola; Jonathan D Silk; Aruna Jeans; Petr A Illarionov; Mariolina Salio; Gurdyal S Besra; Raymond Dwek; Terry D Butters; Frances M Platt; Vincenzo Cerundolo
Journal:  J Exp Med       Date:  2006-09-18       Impact factor: 14.307
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  11 in total

1.  Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice.

Authors:  Albert Ruzo; Miquel Garcia; Albert Ribera; Pilar Villacampa; Virginia Haurigot; Sara Marcó; Eduard Ayuso; Xavier M Anguela; Carles Roca; Judith Agudo; David Ramos; Jesús Ruberte; Fatima Bosch
Journal:  Mol Ther       Date:  2011-10-18       Impact factor: 11.454

2.  Considerations for the treatment of infantile neuronal ceroid lipofuscinosis (infantile Batten disease).

Authors:  Mark S Sands
Journal:  J Child Neurol       Date:  2013-09       Impact factor: 1.987

Review 3.  An emerging phenotype of central nervous system involvement in Pompe disease: from bench to bedside and beyond.

Authors:  Aditi Korlimarla; Jeong-A Lim; Priya S Kishnani; Baodong Sun
Journal:  Ann Transl Med       Date:  2019-07

4.  Lessons learnt from animal models: pathophysiology of neuropathic lysosomal storage disorders.

Authors:  Kim M Hemsley; John J Hopwood
Journal:  J Inherit Metab Dis       Date:  2010-05-07       Impact factor: 4.982

5.  Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII.

Authors:  Ha T Huynh; Jeffrey H Grubb; Carole Vogler; William S Sly
Journal:  Proc Natl Acad Sci U S A       Date:  2012-10-01       Impact factor: 11.205

6.  Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy.

Authors:  Elizabeth Y Qin; Jacqueline A Hawkins-Salsbury; Xuntian Jiang; Adarsh S Reddy; Nuri B Farber; Daniel S Ory; Mark S Sands
Journal:  Mol Genet Metab       Date:  2012-06-01       Impact factor: 4.797

Review 7.  Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease).

Authors:  Jacqueline A Hawkins-Salsbury; Jonathan D Cooper; Mark S Sands
Journal:  Biochim Biophys Acta       Date:  2013-06-06

Review 8.  Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.

Authors:  Shannon L Macauley
Journal:  Pediatr Endocrinol Rev       Date:  2016-06

9.  Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage disease.

Authors:  Yong Hong Chen; Kristin Claflin; James C Geoghegan; Beverly L Davidson
Journal:  Mol Ther       Date:  2012-05-15       Impact factor: 11.454

10.  Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis.

Authors:  Su Xu; Lingling Wang; Mukarram El-Banna; Istvan Sohar; David E Sleat; Peter Lobel
Journal:  Mol Ther       Date:  2011-07-05       Impact factor: 12.910
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