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Development of a fibrin-mediated gene delivery system for the treatment of cystinosis via design of experiment
Cystinosis is a rare disease, caused by a mutation in the gene cystinosin and characterised by the accumulation of cystine crystals. Advantages of biomaterial-mediated gene delivery include reduced safety concerns and the possibility to cure organs that are difficult to treat using systemic gene tra...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Nature Publishing Group UK
2022
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8904479/ https://www.ncbi.nlm.nih.gov/pubmed/35260693 http://dx.doi.org/10.1038/s41598-022-07750-y |