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Gene therapy for the neurological manifestations in lysosomal storage disorders
Over the past several years, considerable progress has been made in the development of gene therapy as a therapeutic strategy for a variety of inherited metabolic diseases, including neuropathic lysosomal storage disorders (LSDs). The premise of gene therapy for this group of diseases is borne of fi...
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| Udgivet i: | J Lipid Res |
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| Hovedforfatter: | |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
The American Society for Biochemistry and Molecular Biology
2014
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4617364/ https://ncbi.nlm.nih.gov/pubmed/24683200 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1194/jlr.R047175 |
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