ArticleStem cells translational medicine2017
Efficiently Specified Ventral Midbrain Dopamine Neurons from Human Pluripotent Stem Cells Under Xeno-Free Conditions Restore Motor Deficits in Parkinsonian Rodents.
Article in Stem cells translational medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 42 papers.
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Who cites it
42 citing papers in PubMed, 70 citations in OpenAlex.
- Cryopreservable dopaminergic progenitors derived from human iPSCs with accelerated loss of pluripotency and early functional restoration in Parkinsonian rats.NPJ Parkinson's disease · 2026Article
- The immunological profile of RC17 hESC-derived dopaminergic neural progenitor cells in vitro: Implications for the STEM-PD clinical trial.Stem cell reports · 2026Article
- Harnessing Intelligence from Brain Cells In Vitro.The Neuroscientist : a review journal bringing neurobiology, neurology and psychiatry · 2025Review
- Exercise promotes the functional integration of human stem cell-derived neural grafts in a rodent model of Parkinson's disease.Stem cell reports · 2025Article
- Assembly of a stem cell-derived human postimplantation embryo model.Nature protocols · 2025Article
- Discovery of NRG1-VII: the myeloid-derived class of NRG1.BMC genomics · 2024Article
- Past, present, and future of cell replacement therapy for parkinson's disease: a novel emphasis on host immune responses.Cell research · 2024Review
- Multiple factors to assist human-derived induced pluripotent stem cells to efficiently differentiate into midbrain dopaminergic neurons.Neural regeneration research · 2024Article
- High Yield of Functional Dopamine-like Neurons Obtained in NeuroForsk 2.0 Medium to Study Acute and Chronic Rotenone Effects on Oxidative Stress, Autophagy, and Apoptosis.International journal of molecular sciences · 2023Article
- Directional induction of neural stem cells, a new therapy for neurodegenerative diseases and ischemic stroke.Cell death discovery · 2023Review
- Identification of cell barcodes from long-read single-cell RNA-seq with BLAZE.Genome biology · 2023Article
- Challenges in the clinical advancement of cell therapies for Parkinson's disease.Nature biomedical engineering · 2023Review
- Biochemical Characteristics of iPSC-Derived Dopaminergic Neurons from N370SInternational journal of molecular sciences · 2023Article
- From 2D to 3D: Development of Monolayer Dopaminergic Neuronal and Midbrain Organoid Cultures for Parkinson's Disease Modeling and Regenerative Therapy.International journal of molecular sciences · 2023Review
- TGFβ3, dibutyryl cAMP and a notch inhibitor modulate phenotype late in stem cell-derived dopaminergic neuron maturation.Frontiers in cell and developmental biology · 2023Article
- Challenges involved in cell therapy for Parkinson's disease using human pluripotent stem cells.Frontiers in cell and developmental biology · 2023Review
- Pretreatment with Perlecan-Conjugated Laminin-E8 Fragment Enhances Maturation of Grafted Dopaminergic Progenitors in Parkinson's Disease Model.Stem cells translational medicine · 2022Article
- Understanding the Influence of Target Acquisition on Survival, Integration, and Phenotypic Maturation of Dopamine Neurons within Stem Cell-Derived Neural Grafts in a Parkinson's Disease Model.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2022Article
- Robust derivation of transplantable dopamine neurons from human pluripotent stem cells by timed retinoic acid delivery.Nature communications · 2022Article
- Optimizing maturity and dose of iPSC-derived dopamine progenitor cell therapy for Parkinson's disease.NPJ Regenerative medicine · 2022Article
Corrections and comments
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Authors and funding
11 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Recent studies have shown evidence for the functional integration of human pluripotent stem cell (hPSC)-derived ventral midbrain dopamine (vmDA) neurons in animal models of Parkinson's disease. Although these cells present a sustainable alternative to fetal mesencephalic grafts, a number of hurdles require attention prior to clinical translation. These include the persistent use of xenogeneic reagents and challenges associated with scalability and storage of differentiated cells. In this study, we describe the first fully defined feeder- and xenogeneic-free protocol for the generation of vmDA neurons from hPSCs and utilize two novel reporter knock-in lines (LMX1A-eGFP and PITX3-eGFP) for in-depth in vitro and in vivo tracking. Across multiple embryonic and induced hPSC lines, this "next generation" protocol consistently increases both the yield and proportion of vmDA neural progenitors (OTX2/FOXA2/LMX1A) and neurons (FOXA2/TH/PITX3) that display classical vmDA metabolic and electrophysiological properties. We identify the mechanism underlying these improvements and demonstrate clinical applicability with the first report of scalability and cryopreservation of bona fide vmDA progenitors at a time amenable to transplantation. Finally, transplantation of xeno-free vmDA progenitors from LMX1A- and PITX3-eGFP reporter lines into Parkinsonian rodents demonstrates improved engraftment outcomes and restoration of motor deficits. These findings provide important and necessary advancements for the translation of hPSC-derived neurons into the clinic. Stem Cells Translational Medicine 2017;6:937-948.
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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.