Literature DB >> 18341388

Central nervous system therapy for lysosomal storage disorders.

Gregory M Enns1, Stephen L Huhn.   

Abstract

Most lysosomal storage disorders are characterized by progressive central nervous system impairment, with or without systemic involvement. Affected individuals have an array of symptoms related to brain dysfunction, the most devastating of which is neurodegeneration following a period of normal development. The blood-brain barrier has represented a significant impediment to developing therapeutic approaches to treat brain disease, but novel approaches-including enzyme replacement, small-molecule, gene, and cell-based therapies-have given children afflicted by these conditions and those who care for them hope for the future.

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Year:  2008        PMID： 18341388     DOI： 10.3171/FOC/2008/24/3-4/E11

Source DB:  PubMed          Journal:  Neurosurg Focus        ISSN： 1092-0684            Impact factor:   4.047


  17 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.  Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients.

Authors:  Moin Vera; Steven Le; Shih-Hsin Kan; Hermes Garban; David Naylor; Anton Mlikotic; Ilkka Kaitila; Paul Harmatz; Agnes Chen; Patricia Dickson
Journal:  Pediatr Res       Date:  2013-09-03       Impact factor: 3.756

3.  Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.

Authors:  Diane Golebiowski; Imramsjah M J van der Bom; Churl-Su Kwon; Andrew D Miller; Keiko Petrosky; Allison M Bradbury; Stacy Maitland; Anna Luisa Kühn; Nina Bishop; Elizabeth Curran; Nilsa Silva; Dwijit GuhaSarkar; Susan V Westmoreland; Douglas R Martin; Matthew J Gounis; Wael F Asaad; Miguel Sena-Esteves
Journal:  Hum Gene Ther       Date:  2017-01-26       Impact factor: 5.695

Review 4.  Potential use of stem cells as a therapy for cystinosis.

Authors:  Celine J Rocca; Stephanie Cherqui
Journal:  Pediatr Nephrol       Date:  2018-05-22       Impact factor: 3.714

Review 5.  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

Review 6.  Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.

Authors:  Alessandra Biffi
Journal:  Mol Ther       Date:  2017-04-04       Impact factor: 11.454

7.  CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome).

Authors:  Sandra Motas; Virginia Haurigot; Miguel Garcia; Sara Marcó; Albert Ribera; Carles Roca; Xavier Sánchez; Víctor Sánchez; Maria Molas; Joan Bertolin; Luca Maggioni; Xavier León; Jesús Ruberte; Fatima Bosch
Journal:  JCI Insight       Date:  2016-06-16

8.  Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy.

Authors:  Virginia Haurigot; Sara Marcó; Albert Ribera; Miguel Garcia; Albert Ruzo; Pilar Villacampa; Eduard Ayuso; Sònia Añor; Anna Andaluz; Mercedes Pineda; Gemma García-Fructuoso; Maria Molas; Luca Maggioni; Sergio Muñoz; Sandra Motas; Jesús Ruberte; Federico Mingozzi; Martí Pumarola; Fatima Bosch
Journal:  J Clin Invest       Date:  2013-07-01       Impact factor: 14.808

9.  High-dose enzyme replacement therapy in murine Hurler syndrome.

Authors:  Li Ou; Tyler Herzog; Brenda L Koniar; Roland Gunther; Chester B Whitley
Journal:  Mol Genet Metab       Date:  2013-09-19       Impact factor: 4.797

Review 10.  Stem Cells in Neurological Disorders: Emerging Therapy with Stunning Hopes.

Authors:  Ghanshyam Upadhyay; Sharmila Shankar; Rakesh K Srivastava
Journal:  Mol Neurobiol       Date:  2014-09-23       Impact factor: 5.590
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