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Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases
More than 50 lysosomal storage diseases (LSDs) are associated with lysosomal dysfunctions with the frequency of 1:5,000 live births. As a result of missing enzyme activity, the lysosome dysfunction accumulates undegraded or partially degraded molecules, affecting the entire body. Most of them are li...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2021
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8688940/ https://www.ncbi.nlm.nih.gov/pubmed/34977274 http://dx.doi.org/10.1016/j.omtm.2021.11.007 |