ArticleCell stem cell2021
Preclinical Efficacy and Safety of a Human Embryonic Stem Cell-Derived Midbrain Dopamine Progenitor Product, MSK-DA01.
Article in Cell stem cell, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 130 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
130 citing papers in PubMed, 3 syntheses or guidelines pooled it, 244 citations in OpenAlex.
- Therapeutic Potential of PSC-derived Cell Transplantation in Parkinson's Disease: A Systematic Review and Meta-analysis of Preclinical Studies.Current stem cell research & therapy · 2026Pooled it
- Research trends on neuroinflammation in Parkinson's disease: an exploratory analysis.Frontiers in aging neuroscience · 2026Pooled it
- Building on a Solid Foundation: Adding Relevance and Reproducibility to Neurological Modeling Using Human Pluripotent Stem Cells.Frontiers in cellular neuroscience · 2021Pooled it
- The TransEuro open-label trial of human fetal ventral mesencephalic transplantation in patients with moderate Parkinson's disease.Nature biotechnology · 2026Trial
- Trial
- Direct neuronal reprogramming: emerging therapeutic strategies for neurodegenerative disorders.Annals of medicine · 2026Review
- Brain organoids as models of extracellular vesicle-mediated human neural communication.Neural regeneration research · 2026Article
- Review
- Beyond phenotypic markers: rethinking dopaminergic identity in iPSC-derived neurons.Cell communication and signaling : CCS · 2026Review
- Parkinson's disease as a multi-axis systems disorder: integrating molecular pathology and circuit-level therapeutics.Cell communication and signaling : CCS · 2026Review
- Optimized Midbrain Organoid Transplantation Restores Dopaminergic Nigrostriatal Pathway and Motor Function in Parkinson's Disease Mouse Model.Neuroscience bulletin · 2026Article
- iSCORE-PD: an isogenic stem cell collection to research Parkinson's disease.Nature communications · 2026Article
- Rapid and Robust Generation of Human Cortical Interneurons from Induced Neural Stem Cells.International journal of molecular sciences · 2026Article
- Stem Cell Therapy for Parkinson's Disease: A Mechanistically Distinct Role for Muse Cells.Journal of clinical medicine · 2026Review
- Cryopreservable dopaminergic progenitors derived from human iPSCs with accelerated loss of pluripotency and early functional restoration in Parkinsonian rats.NPJ Parkinson's disease · 2026Article
- Modulation of WNT and FGF18 enhances yield and subtype identity of hPSC-derived midbrain dopamine neurons.The Journal of clinical investigation · 2026Article
- Review
- Human pluripotent stem cell engineering with CRISPR-Cas9 for Parkinson's disease.Experimental & molecular medicine · 2026Review
- Organoid Technology as a Promising Frontier in Organ Transplantation and Regenerative Medicine.International journal of stem cells · 2026Review
- Telomerase-active urine-derived stem cells: Regenerative solutions for aging.Ageing research reviews · 2026Review
70 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
24 authors at 2 institutions in 1 country.
Funding
Abstract
Parkinson's disease is characterized by the loss of dopaminergic neurons in the substantia nigra leading to disabling deficits. Dopamine neuron grafts may provide a significant therapeutic advance over current therapies. We have generated midbrain dopamine neurons from human embryonic stem cells and manufactured large-scale cryopreserved dopamine progenitors for clinical use. After optimizing cell survival and phenotypes in short-term studies, the cell product, MSK-DA01, was subjected to an extensive set of biodistribution, toxicity, and tumorigenicity assessments in mice under GLP conditions. A large-scale efficacy study was also performed in rats with the same lot of cells intended for potential human use and demonstrated survival of the grafted cells and behavioral amelioration in 6-hydroxydopamine lesioned rats. There were no adverse effects attributable to the grafted cells, no obvious distribution outside the brain, and no cell overgrowth or tumor formation, thus paving the way for a future clinical trial.
Indexed as
Identifiers
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.