Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

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

Journal of the American Society of Nephrology : JASN - 1 May 2020

Hollywood Jennifer A, Przepiorski Aneta, D'Souza Randall F, Sreebhavan Sreevalsan, Wolvetang Ernst J, Harrison Patrick T, Davidson Alan J, Holm Teresa M

Abstract excerpt

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, proliferation, survival, and autophagy. In its severest form, cystinosis is characterized by cystine accumulation, renal...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Amino Acid Transport Systems, Neutral
Share to Cystine

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Cystinosis

Share to Drug Evaluation, Preclinical

Share to Everolimus

Share to Heterografts

Share to Organoids

Share to TOR Serine-Threonine Kinases

Source

Open in PubMed Open full text

Identifiers

PMID: 32198276
DOI: 10.1681/ASN.2019070712
PMCID: PMC7217405

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Use of Human Induced Pluripotent Stem Cells and Kidney Organoids To Develop a Cysteamine/mTOR Inhibition Combination Therapy for Cystinosis.PMID 32198276Read ↗

Use of human iPSCs and kidney organoids to develop a cysteamine/mTOR inhibition combination therapy to treat cystinosisSemantic proximity Read ↗

Molecular Mechanisms and Treatment Options of Nephropathic Cystinosis.Semantic proximity Read ↗

Cysteamine-bicalutamide combination therapy corrects proximal tubule phenotype in cystinosis.Semantic proximity Read ↗

Cysteamine-bicalutamide combination treatment restores alpha-ketoglutarate and corrects proximal tubule phenotype in cystinosisSemantic proximity Read ↗

Hematopoietic Stem Cell Gene Therapy for Cystinosis: From Bench-to-BedsideSemantic proximity Read ↗

Bioengineered Cystinotic Kidney Tubules Recapitulate a Nephropathic Phenotype.Semantic proximity Read ↗

In Vitro and In Vivo Models to Study Nephropathic Cystinosis.Semantic proximity Read ↗

Cystinosis.Semantic proximity Read ↗

Gene surgery as a potential treatment option for Nephropathic Cystinosis <i>in vitro</i>Semantic proximity Read ↗

Cell-Based Phenotypic Drug Screening Identifies Luteolin as Candidate Therapeutic for Nephropathic Cystinosis.Semantic proximity Read ↗

SGLT2 Inhibitors Rescue Lysosomal mTORC1 Hyperactivity and Proximal Tubulopathy in Preclinical Models of CystinosisSemantic proximity Read ↗

Nephropathic cystinosis: an update on genetic conditioning.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.