Literature DB >> 16049428

Storage solutions: treating lysosomal disorders of the brain.

Mylvaganam Jeyakumar1, Raymond A Dwek, Terry D Butters, Frances M Platt.   

Abstract

Many neurodegenerative diseases are characterized by the accumulation of undegradable molecules in cells or at extracellular sites in the brain. One such family of diseases is the lysosomal storage disorders, which result from defects in various aspects of lysosomal function. Until recently, there was little prospect of treating storage diseases involving the CNS. However, recent progress has been made in understanding these conditions and in translating the findings into experimental therapies. We review the developments in this field and discuss the similarities in pathological features between these diseases and some more common neurodegenerative disorders.

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Year:  2005        PMID: 16049428     DOI: 10.1038/nrn1725

Source DB:  PubMed          Journal:  Nat Rev Neurosci        ISSN: 1471-003X            Impact factor:   34.870


  78 in total

Review 1.  Degradation of connexins through the proteasomal, endolysosomal and phagolysosomal pathways.

Authors:  Vivian Su; Kimberly Cochrane; Alan F Lau
Journal:  J Membr Biol       Date:  2012-07-08       Impact factor: 1.843

Review 2.  Pathophysiology of neuropathic lysosomal storage disorders.

Authors:  Cinzia Maria Bellettato; Maurizio Scarpa
Journal:  J Inherit Metab Dis       Date:  2010-04-29       Impact factor: 4.982

Review 3.  Common and uncommon pathogenic cascades in lysosomal storage diseases.

Authors:  Einat B Vitner; Frances M Platt; Anthony H Futerman
Journal:  J Biol Chem       Date:  2010-04-29       Impact factor: 5.157

4.  Nanomedicine in the diagnosis and therapy of neurodegenerative disorders.

Authors:  A V Kabanov; H E Gendelman
Journal:  Prog Polym Sci       Date:  2007       Impact factor: 29.190

5.  Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders.

Authors:  Silvia Muro
Journal:  Drug Deliv Transl Res       Date:  2012-06-01       Impact factor: 4.617

6.  Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy.

Authors:  John Marshall; Kerry Anne McEachern; Wei-Lien Chuang; Elizabeth Hutto; Craig S Siegel; James A Shayman; Greg A Grabowski; Ronald K Scheule; Diane P Copeland; Seng H Cheng
Journal:  J Inherit Metab Dis       Date:  2010-03-25       Impact factor: 4.982

7.  Midbody ring disposal by autophagy is a post-abscission event of cytokinesis.

Authors:  Christian Pohl; Stefan Jentsch
Journal:  Nat Cell Biol       Date:  2008-12-14       Impact factor: 28.824

8.  The effects of early and late bone marrow transplantation in siblings with alpha-mannosidosis. Is early haematopoietic cell transplantation the preferred treatment option?

Authors:  A A Broomfield; A Chakrapani; J E Wraith
Journal:  J Inherit Metab Dis       Date:  2010-02-18       Impact factor: 4.982

9.  Deletion in the N-terminal half of olfactomedin 1 modifies its interaction with synaptic proteins and causes brain dystrophy and abnormal behavior in mice.

Authors:  Naoki Nakaya; Afia Sultana; Jeeva Munasinghe; Aiwu Cheng; Mark P Mattson; Stanislav I Tomarev
Journal:  Exp Neurol       Date:  2013-10-02       Impact factor: 5.330

10.  Early changes in the apparent diffusion coefficient (ADC) in a mouse model of Sandhoff's disease occur prior to disease symptoms and behavioral deficits.

Authors:  Lingyun Hu; Yingying Sun; Laura E Villasana; Richard Paylor; Eric Klann; Robia G Pautler
Journal:  Magn Reson Med       Date:  2009-11       Impact factor: 4.668

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