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Chaperone–Mediated Gene Therapy with Recombinant AAV-PPCA in a New Mouse Model of Type I Sialidosis
The lysosomal storage disease sialidosis is caused by a primary deficiency of the sialidase NEU1. Patients with type I sialidosis develop an attenuated, non-neuropathic form of the disease also named cherry red spot myoclonus syndrome, with symptoms arising during juvenile/ adult age. NEU1 requires...
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| Main Authors: | , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2013
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3794473/ https://ncbi.nlm.nih.gov/pubmed/23770387 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bbadis.2013.06.002 |
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