Use of Human Induced Pluripotent Stem Cells and Kidney Organoids To Develop a Cysteamine/mTOR Inhibition Combination Therapy for Cystinosis

BACKGROUND: Mutations in CTNS—a gene encoding the cystine transporter cystinosin—cause the rare, autosomal, recessive, lysosomal-storage disease cystinosis. Research has also implicated cystinosin in modulating the mTORC1 pathway, which serves as a core regulator of cellular metabolism, proliferatio...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:J Am Soc Nephrol
Prif Awduron: Hollywood, Jennifer A., Przepiorski, Aneta, D’Souza, Randall F., Sreebhavan, Sreevalsan, Wolvetang, Ernst J., Harrison, Patrick T., Davidson, Alan J., Holm, Teresa M.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Nephrology 2020
Pynciau:
Basic Research
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC7217405/
https://ncbi.nlm.nih.gov/pubmed/32198276
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1681/ASN.2019070712
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