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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Detalles Bibliográficos
Autor principal: Cherqui, Stephanie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Review
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

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8699556/
https://www.ncbi.nlm.nih.gov/pubmed/34943781
http://dx.doi.org/10.3390/cells10123273

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