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...

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Detalles Bibliográficos
Autores principales: Rintz, Estera, Higuchi, Takashi, Kobayashi, Hiroshi, Galileo, Deni S., Wegrzyn, Grzegorz, Tomatsu, Shunji
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Review
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

Internet

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

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