Cell therapy for cystic fibrosis

Murphy, Sean V.; Atala, Anthony

doi:10.1002/term.1746

Cited by 7 publications

(5 citation statements)

References 139 publications

(170 reference statements)

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“…The current strategy to increase the effectiveness of the treatment could be based on the application of cell therapy. MSCs have a number of unique properties that make them attractive for use in various fields of regenerative medicine, in particular urology (Aboushwareb & Atala, ; Murphy, & Atala, ; Skrahin et al, ; Zhou et al, ). Several hundred clinical trials have used MSCs for the therapy of cancer, autoimmune diseases, and the control of inflammation during organ transplantation.…”

Section: Discussionmentioning

confidence: 99%

Application of the allogenic mesenchymal stem cells in the therapy of the bladder tuberculosis

Yudintceva

Bogolyubova

Muraviov

et al. 2017

J Tissue Eng Regen Med

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Urogenital tuberculosis (TB) often leads to contraction of the bladder, a reduction of the urinary reservoir capacity, and, in the latest stage, to real microcystitis up to full obliteration. Bladder TB Stage 4 is unsuitable for conservative therapy, and cystectomy with subsequent enteroplasty is indicated. In this study, using a model of bladder TB in New Zealand rabbits, the therapeutic efficacy of the interstitial injection of autologous bone-derived mesenchymal stem cells (MSCs) combined with standard anti-TB treatment in the restoration of the bladder function was demonstrated. For analysis of the MSC distribution in tissues, the latter were labelled with superparamagnetic iron oxide nanoparticles. In vitro studies demonstrated the high intracellular incorporation of nanoparticles and the absence of cytotoxicity on MSC viability and proliferation. A single-dose administration of MSCs into the bladder mucosal layer significantly reduced the wall deformation and inflammation and hindered the development of fibrosis, which was proven by the subsequent histological assay. Confocal microscopy studies of the bladder cryosections confirmed the presence of superparamagnetic iron oxide nanoparticle-labelled MSCs in different bladder layers of the treated animals, thus indicating the role of stem cells in bladder regeneration.

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Section: Discussionmentioning

confidence: 99%

Application of the allogenic mesenchymal stem cells in the therapy of the bladder tuberculosis

Yudintceva

Bogolyubova

Muraviov

et al. 2017

J Tissue Eng Regen Med

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“…MSCs also have the ability to act as antigen-presenting cells, modulate the local immune response, and enhance natural repair mechanisms through activation of endogenous progenitors and mature cells 17 . Accordingly, pre-clinical studies of MSC therapy in acute respiratory distress syndrome (ARDS) 18 , cystic fibrosis 19 , and emphysema 20 have revealed potential therapeutic benefits of MSCs in the treatment of these diseases.…”

Section: Introductionmentioning

confidence: 99%

Controlled delivery and minimally invasive imaging of stem cells in the lung

Kim

Guenthart

O’Neill

et al. 2017

Sci Rep

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Intratracheal delivery of stem cells into injured or diseased lungs can provide a variety of therapeutic and immunomodulatory effects for the treatment of acute lung injury and chronic lung disease. While the efficacy of this approach depends on delivering the proper cell dosage into the target region of the airway, tracking and analysis of the cells have been challenging, largely due to the limited understanding of cell transport and lack of suitable cell monitoring techniques. We report on the transport and deposition of intratracheally delivered stem cells as well as strategies to modulate the number of cells (e.g., dose), topographic distribution, and region-specific delivery in small (rodent) and large (porcine and human) lungs. We also developed minimally invasive imaging techniques for real-time monitoring of intratracheally delivered cells. We propose that this approach can facilitate the implementation of patient-specific cells and lead to enhanced clinical outcomes in the treatment of lung disease with cell-based therapies.

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“…Cell therapy could be a more effective treatment because allogeneic normal cells and autologous engineered cells express CFTR gene. Indeed, various cell types have been utilized for treating CF . Bone marrow (BM)‐derived stem cells have been the first source evaluated for homing to the lung and curative potential.…”

Section: Introductionmentioning

confidence: 99%

Correction of defective CFTR/ENaC function and tightness of cystic fibrosis airway epithelium by amniotic mesenchymal stromal (stem) cells

Carbone

Castellani

Favia

et al. 2014

J Cellular Molecular Medi

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Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, with most of the mortality given by the lung disease. Human amniotic mesenchymal stromal (stem) cells (hAMSCs) hold great promise for regenerative medicine in the field of lung disease; however, their potential as therapeutics for CF lung disease has not been fully explored. In the present study, hAMSCs were analysed in co-cultures on Transwell filters with CF immortalized airway epithelial cells (CFBE41o- line) at different ratios to exploit their potency to resume basic defects associated with CF. The results show that F-actin content was increased in co-cultures as compared with CF cells and actin was reorganized to form stress fibres. Confocal microscopy studies revealed that co-cultures had a tendency of increased expression of occludin and ZO-1 at the intercellular borders, paralleled by a decrease in dextran permeability, suggestive of more organized tight junctions (TJs). Spectrofluorometric analysis of CFTR function demonstrated that hAMSC-CFBE co-cultures resumed chloride transport, in line with the appearance of the mature Band C of CFTR protein by Western blotting. Moreover, hAMSC-CFBE co-cultures, at a 1:5 ratio, showed a decrease in fluid absorption, as opposed to CFBE cell monolayers that displayed a great rate of fluid resorption from the apical side. Our data show that human amniotic MSCs can be used in co-culture with CF respiratory epithelial cells to model their engraftment into the airways and have the potential to resume a tight epithelium with partial correction of the CF phenotype.

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Cell therapy for cystic fibrosis

Cited by 7 publications

References 139 publications

Application of the allogenic mesenchymal stem cells in the therapy of the bladder tuberculosis

Application of the allogenic mesenchymal stem cells in the therapy of the bladder tuberculosis

Controlled delivery and minimally invasive imaging of stem cells in the lung

Correction of defective CFTR/ENaC function and tightness of cystic fibrosis airway epithelium by amniotic mesenchymal stromal (stem) cells

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