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Hematopoietic Stem Cell Gene Therapy for Cystinosis: From Bench-to-Bedside
Cystinosis is an autosomal recessive metabolic disease that belongs to the family of lysosomal storage disorders. The gene involved is the CTNS gene that encodes cystinosin, a seven-transmembrane domain lysosomal protein, which is a proton-driven cystine transporter. Cystinosis is characterized by t...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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MDPI
2021
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8699556/ https://www.ncbi.nlm.nih.gov/pubmed/34943781 http://dx.doi.org/10.3390/cells10123273 |