Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice

Metachromatic leukodystrophy (MLD) is a lysosomal storage disorder characterized by accumulation of sulfatides in glial cells and neurons, the result of an inherited deficiency of arylsulfatase A (ARSA; EC 3.1.6.8) and myelin degeneration in the central and peripheral nervous systems. No effective t...

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Hlavní autoři: Piguet, Françoise, Sondhi, Dolan, Piraud, Monique, Fouquet, Françoise, Hackett, Neil R., Ahouansou, Ornella, Vanier, Marie-Thérèse, Bieche, Ivan, Aubourg, Patrick, Crystal, Ronald G., Cartier, Nathalie, Sevin, Caroline
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2012
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Research Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3413898/
https://ncbi.nlm.nih.gov/pubmed/22642214
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.015
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