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

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

Who cites it

17 citing papers in PubMed.

1. Gene therapy for the nervous system: challenges and new strategies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2014
   Review
2. Using ASMase knockout mice to model human diseases.Handbook of experimental pharmacology · 2013
   Review
3. Organelle targeting: third level of drug targeting.Drug design, development and therapy · 2013
   Review
4. Evaluation of neurodegeneration in a mouse model of infantile batten disease by magnetic resonance imaging and magnetic resonance spectroscopy.Neuro-degenerative diseases · 2012
   Article
5. Neural stem cell transplantation as a therapeutic approach for treating lysosomal storage diseases.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2011
   Review
6. Disruption of adaptive energy metabolism and elevated ribosomal p-S6K1 levels contribute to INCL pathogenesis: partial rescue by resveratrol.Human molecular genetics · 2011
   Article
7. Molecular simulations of carbohydrates and protein-carbohydrate interactions: motivation, issues and prospects.Drug discovery today · 2010
   Review
8. Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice.Gene therapy · 2009
   Article
9. Structural glycobiology: a game of snakes and ladders.Glycobiology · 2008
   Review
10. Endocytic mechanisms for targeted drug delivery.Advanced drug delivery reviews · 2007
    Review
11. Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice.Gene therapy · 2007
    Article
12. Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.Molecular therapy : the journal of the American Society of Gene Therapy · 2006
    Article
13. Intracerebral transplantation of adult mouse neural progenitor cells into the Niemann-Pick-A mouse leads to a marked decrease in lysosomal storage pathology.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2004
    Article
14. Deletion of macrophage-inflammatory protein 1 alpha retards neurodegeneration in Sandhoff disease mice.Proceedings of the National Academy of Sciences of the United States of America · 2004
    Article
15. Adeno-associated virus vector-mediated transduction in the cat brain.Gene therapy · 2003
    Article
16. Viral vectors for gene delivery to the nervous system.Nature reviews. Neuroscience · 2003
    Review
17. Nanoparticle transport across the blood brain barrier.Tissue barriers
    Review

Corrections and comments

• Comment on
  Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors.2002

Authors and funding

2 authors.

Funding

No grant is acknowledged in the PubMed record.

Abstract

PubMed holds no abstract for this paper.

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