ArticleStem cells (Dayton, Ohio)2015
Brief reports: Lysosomal cross-correction by hematopoietic stem cell-derived macrophages via tunneling nanotubes.
Article in Stem cells (Dayton, Ohio), 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 80 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 1/2 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34+ Hematopoietic Stem Cells (HSC) From Mobilized Peripheral Blood Stem Cells (PBSC) of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS or pCDY.EFS.CTNS.T260I Lentiviral Vector and Will Include Transduction Enhancer When Required During Manufacturing
A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04
Who cites it
80 citing papers in PubMed, 124 citations in OpenAlex.
- Hematopoietic Stem-Cell Gene Therapy for Cystinosis.The New England journal of medicine · 2026Trial
- Edited HSPCs rescue frataxin deficiency and inflammation-driven multisystemic pathology in Friedreich's ataxia mice.The Journal of clinical investigation · 2026Article
- Cells heal themselves: gene-edited hematopoietic stem cells restore frataxin deficiency in multiple organs.The Journal of clinical investigation · 2026Article
- Tunneling nanotubes: a new dimension of intercellular communication and recent progress.Cell communication and signaling : CCS · 2026Review
- The molecular architecture of tunneling nanotubes.bioRxiv : the preprint server for biology · 2026Article
- Cystinosin regulates NaEMBO reports · 2026Article
- Breaking cellular boundaries: molecular mechanisms of tunneling nanotube formation and fusion.Biochemical Society transactions · 2026Review
- CD13 activation assembles phosphoinositide (PI) signaling complexes to regulate the actin cytoskeleton.Scientific reports · 2026Article
- Nano- and Microtubes: Tiny Fusion Events but Not Overall Plasma Membrane Merger for Exchanging Intracellular Components.Advances in experimental medicine and biology · 2026Review
- Flexible Depth Probes with PEDOT:PSS Microelectrodes for Chronic Recording and Stimulation.ACS applied materials & interfaces · 2025Article
- Induction of tunnelling nanotube-like structures by influenza A viruses requires the onset of apoptosis.PLoS pathogens · 2025Article
- Soma-germ contact across the basement membrane in the ovary.Biology letters · 2025Article
- From Molecular Therapies to Lysosomal Transplantation and Targeted Drug Strategies: Present Applications, Limitations, and Future Prospects of Lysosomal Medications.Biomolecules · 2025Review
- Cystinosin is involved in NabioRxiv : the preprint server for biology · 2025Article
- Emerging therapeutic strategies for cystinosis.Frontiers in pediatrics · 2025Review
- Protective effect of mesenchymal stromal cells in diabetic nephropathy: the In vitro and In vivo role of the M-Sec-tunneling nanotubes.Clinical science (London, England : 1979) · 2024Article
- Review
- A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach.International journal of biological sciences · 2024Review
- Tunneling Nanotubes: The Cables for Viral Spread and Beyond.Results and problems in cell differentiation · 2024Review
- Tunneling Nanotubes in Myeloid Cells: Perspectives for Health and Infectious Diseases.Results and problems in cell differentiation · 2024Review
20 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 3 institutions in 2 countries.
Funding
Abstract
Despite controversies on the potential of hematopoietic stem cells (HSCs) to promote tissue repair, we previously showed that HSC transplantation could correct cystinosis, a multisystemic lysosomal storage disease, caused by a defective lysosomal membrane cystine transporter, cystinosin (CTNS gene). Addressing the cellular mechanisms, we here report vesicular cross-correction after HSC differentiation into macrophages. Upon coculture with cystinotic fibroblasts, macrophages produced tunneling nanotubes (TNTs) allowing transfer of cystinosin-bearing lysosomes into Ctns-deficient cells, which exploited the same route to retrogradely transfer cystine-loaded lysosomes to macrophages, providing a bidirectional correction mechanism. TNT formation was enhanced by contact with diseased cells. In vivo, HSCs grafted to cystinotic kidneys also generated nanotubular extensions resembling invadopodia that crossed the dense basement membranes and delivered cystinosin into diseased proximal tubular cells. This is the first report of correction of a genetic lysosomal defect by bidirectional vesicular exchange via TNTs and suggests broader potential for HSC transplantation for other disorders due to defective vesicular proteins.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.