| Hallett, Penelope J., Deleidi, Michela, Astradsson, Arnar, Smith, Gaynor A.  ORCID: https://orcid.org/0000-0003-4332-8383, Cooper, Oliver, Osborn, Teresia M., Sundberg, Maria, Moore, Michele A., Perez-Torres, Eduardo, Brownell, Anna-Liisa, Schumacher, James M., Spealman, Roger D. and Isacson, Ole
      2015.
      
      Successful function of Autologous iPSC-derived dopamine neurons following transplantation in a non-human primate model of Parkinson's disease.
      Cell Stem Cell
      16
      
        (3)
      
      , pp. 269-274.
      
      10.1016/j.stem.2015.01.018 | 
Abstract
Autologous transplantation of patient-specific induced pluripotent stem cell (iPSC)-derived neurons is a potential clinical approach for treatment of neurological disease. Preclinical demonstration of long-term efficacy, feasibility, and safety of iPSC-derived dopamine neurons in non-human primate models will be an important step in clinical development of cell therapy. Here, we analyzed cynomolgus monkey (CM) iPSC-derived midbrain dopamine neurons for up to 2 years following autologous transplantation in a Parkinson’s disease (PD) model. In one animal, with the most successful protocol, we found that unilateral engraftment of CM-iPSCs could provide a gradual onset of functional motor improvement contralateral to the side of dopamine neuron transplantation, and increased motor activity, without a need for immunosuppression. Postmortem analyses demonstrated robust survival of midbrain-like dopaminergic neurons and extensive outgrowth into the transplanted putamen. Our proof of concept findings support further development of autologous iPSC-derived cell transplantation for treatment of PD.
| Item Type: | Article | 
|---|---|
| Date Type: | Publication | 
| Status: | Published | 
| Schools: | Schools > Medicine | 
| Publisher: | Elsevier | 
| ISSN: | 1934-5909 | 
| Date of Acceptance: | 30 January 2015 | 
| Last Modified: | 03 Nov 2022 09:51 | 
| URI: | https://orca.cardiff.ac.uk/id/eprint/106221 | 
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