Deficiency of the sedoheptulose kinase (Shpk) does not alter the ability of hematopoietic stem cells to rescue cystinosis in the mouse model

Cystinosis is an autosomal recessive lysosomal storage disorder caused by mutations in the CTNS gene encoding the lysosomal cystine transporter, cystinosin, and leading to multi-organ degeneration including kidney failure. A clinical trial for cystinosis is ongoing to test the safety and efficacy of...

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Detalles Bibliográficos
Autores principales: Goodman, Spencer, Khan, Meisha, Sharma, Jay, Li, Zijie, Cano, Jose, Castellanos, Carlos, Estrada, Monica V., Gertsman, Ilya, Cherqui, Stephanie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8935660/
https://www.ncbi.nlm.nih.gov/pubmed/34823997
http://dx.doi.org/10.1016/j.ymgme.2021.11.006

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8935660/
https://www.ncbi.nlm.nih.gov/pubmed/34823997
http://dx.doi.org/10.1016/j.ymgme.2021.11.006

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