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References from From patient cells to patient therapy: Twenty years of iPSC-based translation in neurodegenerative disease. Local targets link to admitted publications; unresolved targets remain external evidence.
Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors
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Pluripotent stem cells in disease modelling and drug discovery
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The future of stem cell therapies for Parkinson disease
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Cell therapy for neurological disorders
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Advancing cell therapy for neurodegenerative diseases
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Fetal dopaminergic transplantation trials and the future of neural grafting in Parkinson's disease
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Replacing Dopamine Neurons in Parkinson's Disease: How did it happen?
10.3233/jpd-179002 · 2017 · External reference
Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson's Disease
10.1056/nejmoa1915872 · 2020 · External reference
Phase I/II trial of iPS-cell-derived dopaminergic cells for Parkinson's disease
10.1038/s41586-025-08700-0 · 2025 · External reference
Phase I trial of hES cell-derived dopaminergic neurons for Parkinson's disease
10.1038/s41586-025-08845-y · 2025 · External reference
Phase 1/2a clinical trial of hESC-derived dopamine progenitors in Parkinson's disease
10.1016/j.cell.2025.09.010 · 2025 · External reference
Human embryonic stem cell-derived dopaminergic cells for Parkinson’s disease: a phase 1/2 open-label trial
10.1038/s41591-026-04525-0 · 2026 · External reference
Translating cell therapies for neurodegenerative diseases: Huntington's disease as a model disorder
10.1093/brain/awac086 · 2022 · External reference
Human stem cell transplantation models of Alzheimer's disease
10.3389/fnagi.2024.1354164 · 2024 · External reference
Non-neuronal cells in amyotrophic lateral sclerosis - from pathogenesis to biomarkers
10.1038/s41582-021-00487-8 · 2021 · External reference
A step closer to autologous cell therapy for Parkinson's disease
10.1016/j.stem.2021.03.010 · 2021 · External reference
Stem cells for the treatment of neurological disorders
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Medium spiny neurons for transplantation in Huntington's disease
10.1042/bst0370323 · 2009 · External reference
Challenges in progressing cell therapies to the clinic for Huntington's disease: A review of the progress made with pluripotent stem cell derived medium spiny neurons
2022 · External reference
hESC-derived striatal progenitors grafted into a Huntington's disease rat model support long-term functional motor recovery by differentiating, self-organizing and connecting into the lesioned striatum
10.1186/s13287-023-03422-4 · 2023 · External reference
Modeling Alzheimer's disease with iPSC-derived brain cells
10.1038/s41380-019-0468-3 · 2020 · External reference
Human iPSC-derived neural progenitor cells secreting GDNF provide protection in rodent models of ALS and retinal degeneration
10.1016/j.stemcr.2023.03.016 · 2023 · External reference
Hypoimmunogenic derivatives of induced pluripotent stem cells evade immune rejection in fully immunocompetent allogeneic recipients
10.1038/s41587-019-0016-3 · 2019 · External reference
Human striatal progenitor cells that contain inducible safeguards and overexpress BDNF rescue Huntington's disease phenotypes
10.1016/j.omtm.2025.101415 · 2025 · External reference
Viral Delivery of GDNF Promotes Functional Integration of Human Stem Cell Grafts in Parkinson's Disease
10.1016/j.stem.2020.01.010 · 2020 · External reference
The history and status of dopamine cell therapies for Parkinson's disease
10.1002/bies.202400118 · 2024 · External reference
Dopamine Cell Therapy: From Cell Replacement to Circuitry Repair
10.3233/jpd-212609 · 2021 · External reference
Grafts of fetal dopamine neurons survive and improve motor function in Parkinson's disease
10.1126/science.2105529 · 1990 · External reference
Randomized Delayed-Start Trial of Levodopa in Parkinson's Disease
10.1056/nejmoa1809983 · 2019 · External reference
A randomized trial of deep-brain stimulation for Parkinson's disease
10.1056/nejmoa060281 · 2006 · External reference
Bilateral deep brain stimulation vs best medical therapy for patients with advanced Parkinson disease: a randomized controlled trial
10.1001/jama.2008.929 · 2009 · External reference
Transplantation of embryonic dopamine neurons for severe Parkinson's disease
10.1056/nejm200103083441002 · 2001 · External reference
A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease
10.1002/ana.10720 · 2003 · External reference
Pre-clinical study of induced pluripotent stem cell-derived dopaminergic progenitor cells for Parkinson's disease
10.1038/s41467-020-17165-w · 2020 · External reference
Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD
10.1016/j.stem.2023.08.014 · 2023 · External reference
Preclinical Efficacy and Safety of a Human Embryonic Stem Cell-Derived Midbrain Dopamine Progenitor Product, MSK-DA01
10.1016/j.stem.2021.01.004 · 2021 · External reference
Human autologous iPSC-derived dopaminergic progenitors restore motor function in Parkinson's disease models
10.1172/jci130767 · 2020 · External reference
Pre-clinical safety and efficacy of human induced pluripotent stem cell-derived products for autologous cell therapy in Parkinson's disease
10.1016/j.stem.2025.01.006 · 2025 · External reference
exPDite-2: A Randomized, Sham-Controlled, Double-Blind Study to Evaluate Bemdaneprocel, a Midbrain Dopaminergic Neuron Progenitor Cell Therapy for Parkinson’s Disease
10.1016/j.parkreldis.2025.107622 · 2025 · External reference
Huntington disease
10.1038/nrdp.2015.5 · 2015 · External reference
The choreography of neuroinflammation in Huntington's disease
10.1016/j.it.2015.04.007 · 2015 · External reference
Effect of Deutetrabenazine on Chorea Among Patients With Huntington Disease: A Randomized Clinical Trial
10.1001/jama.2016.8655 · 2016 · External reference
Tominersen in Adults with Manifest Huntington's Disease
10.1056/nejmc2300400 · 2023 · External reference
Human iPSC-derived neural precursor cells differentiate into multiple cell types to delay disease progression following transplantation into YAC128 Huntington's disease mouse model
10.1111/cpr.13082 · 2021 · External reference
Cell therapy in Huntington’s disease: Taking stock of past studies to move the field forward
10.1002/stem.3300 · 2021 · External reference
Mutant huntingtin is present in neuronal grafts in Huntington disease patients
10.1002/ana.24174 · 2014 · External reference
Motor and cognitive improvements in patients with Huntington's disease after neural transplantation
10.1016/s0140-6736(00)03310-9 · 2000 · External reference
on behalf the Multicentric Intracerebral Grafting in Huntington’s Disease, G. Human Fetal Cell Therapy in Huntington’s Disease: A Randomized, Multicenter, Phase II Trial
10.1002/mds.28201 · 2020 · External reference
Activin A directs striatal projection neuron differentiation of human pluripotent stem cells
10.1242/dev.117093 · 2015 · External reference
Stem Cell-Derived Human Striatal Progenitors Innervate Striatal Targets and Alleviate Sensorimotor Deficit in a Rat Model of Huntington Disease
10.1016/j.stemcr.2020.03.018 · 2020 · External reference
Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells
10.1016/j.stemcr.2017.01.022 · 2017 · External reference
Alzheimer Disease: An Update on Pathobiology and Treatment Strategies
10.1016/j.cell.2019.09.001 · 2019 · External reference
Alzheimer's disease is a synaptic failure
10.1126/science.1074069 · 2002 · External reference
The Cellular Phase of Alzheimer's Disease
10.1016/j.cell.2015.12.056 · 2016 · External reference
Alzheimer's disease and senile dementia: loss of neurons in the basal forebrain
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Inhibitory interneuron deficit links altered network activity and cognitive dysfunction in Alzheimer model
10.1016/j.cell.2012.02.046 · 2012 · External reference
Single-cell multiregion dissection of Alzheimer's disease
10.1038/s41586-024-07606-7 · 2024 · External reference
The global macroeconomic burden of Alzheimer's disease and other dementias: estimates and projections for 152 countries or territories
10.1016/s2214-109x(24)00264-x · 2024 · External reference
Lecanemab in Early Alzheimer's Disease
10.1056/nejmoa2212948 · 2023 · External reference
Donanemab in Early Symptomatic Alzheimer Disease: The TRAILBLAZER-ALZ 2 Randomized Clinical Trial
10.1001/jama.2023.13239 · 2023 · External reference
Harnessing human iPSC-microglia for CNS-wide delivery of disease-modifying proteins
10.1016/j.stem.2025.03.009 · 2025 · External reference
Neural stem cells improve cognition via BDNF in a transgenic model of Alzheimer disease
10.1073/pnas.0901402106 · 2009 · External reference
Induced Pluripotent Stem Cell-Derived Neural Precursors Improve Memory, Synaptic and Pathological Abnormalities in a Mouse Model of Alzheimer's Disease
10.3390/cells10071802 · 2021 · External reference
iPSC-Derived Human Microglia-like Cells to Study Neurological Diseases
10.1016/j.neuron.2017.03.042 · 2017 · External reference
Human iPSC-derived astrocytes transplanted into the mouse brain undergo morphological changes in response to amyloid-beta plaques
10.1186/s13024-021-00487-8 · 2021 · External reference
Gain of BDNF Function in Engrafted Neural Stem Cells Promotes the Therapeutic Potential for Alzheimer's Disease
2016 · External reference
Alzheimer's disease
10.1016/s0140-6736(20)32205-4 · 2021 · External reference
Neural stem cell therapy for neurodegenerative disorders: The role of neurotrophic support
10.1016/j.neuint.2017.02.006 · 2017 · External reference
Finding Treatment Effects in Alzheimer Trials in the Face of Disease Progression Heterogeneity
10.1212/wnl.0000000000012022 · 2021 · External reference
Human stem cell models of neurodegeneration: From basic science of amyotrophic lateral sclerosis to clinical translation
10.1016/j.stem.2021.12.008 · 2022 · External reference
Amyotrophic lateral sclerosis
2017 · External reference
Onset and progression in inherited ALS determined by motor neurons and microglia
10.1126/science.1123511 · 2006 · External reference
Degeneration and impaired regeneration of gray matter oligodendrocytes in amyotrophic lateral sclerosis
10.1038/nn.3357 · 2013 · External reference
Stem cell transplantation for motor neuron disease: current approaches and future perspectives
10.1007/s13311-011-0068-7 · 2011 · External reference
Intraspinal stem cell transplantation for amyotrophic lateral sclerosis
10.1002/ana.24584 · 2016 · External reference
The past, present and future of stem cell clinical trials for ALS
10.1016/j.expneurol.2014.02.021 · 2014 · External reference
Amyotrophic Lateral Sclerosis: A Review
10.1001/jama.2026.6385 · 2026 · External reference
Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis
10.1001/jamaneurol.2025.4946 · 2026 · External reference
Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial
10.1038/s41591-022-01956-3 · 2022 · External reference
Focal transplantation-based astrocyte replacement is neuroprotective in a model of motor neuron disease
10.1038/nn.2210 · 2008 · External reference
Non-cell autonomous effect of glia on motor neurons in an embryonic stem cell-based ALS model
10.1038/nn1885 · 2007 · External reference
Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons
10.1038/nn1876 · 2007 · External reference
Lumbar intraspinal injection of neural stem cells in patients with amyotrophic lateral sclerosis: results of a phase I trial in 12 patients
10.1002/stem.1079 · 2012 · External reference
Human neural stem cell transplantation in ALS: initial results from a phase I trial
10.1186/s12967-014-0371-2 · 2015 · External reference
Current Status and Future Perspectives on Stem Cell-Based Therapies for Parkinson's Disease
10.14802/jmd.22141 · 2023 · External reference
MHC matching improves engraftment of iPSC-derived neurons in non-human primates
10.1038/s41467-017-00926-5 · 2017 · External reference
Engineering universal cells that evade immune detection
10.1038/s41577-019-0200-1 · 2019 · External reference
Control of immune response in an iPSC-based allogeneic cell therapy clinical trial for Parkinson's disease
10.1016/j.stem.2025.07.012 · 2025 · External reference
Innate and adaptive immune responses in the CNS
10.1016/s1474-4422(15)00141-6 · 2015 · External reference
CNS infection and immune privilege
10.1038/s41583-018-0070-8 · 2018 · External reference
Co-transplantation of autologous T(reg) cells in a cell therapy for Parkinson's disease
10.1038/s41586-023-06300-4 · 2023 · External reference
TNF-NF-kappaB-p53 axis restricts in vivo survival of hPSC-derived dopamine neurons
10.1016/j.cell.2024.05.030 · 2024 · External reference
How to make a midbrain dopaminergic neuron
10.1242/dev.097394 · 2015 · External reference
Maintaining differentiated cellular identity
10.1038/nrg3209 · 2012 · External reference
Dopamine neurons derived from human ES cells efficiently engraft in animal models of Parkinson's disease
10.1038/nature10648 · 2011 · External reference
Embryonic stem cell-derived Pitx3-enhanced green fluorescent protein midbrain dopamine neurons survive enrichment by fluorescence-activated cell sorting and function in an animal model of Parkinson’s disease
10.1634/stemcells.2007-0996 · 2008 · External reference
Neural precursors derived from embryonic stem cells, but not those from fetal ventral mesencephalon, maintain the potential to differentiate into dopaminergic neurons after expansion in vitro
10.1634/stemcells.2005-0558 · 2006 · External reference
ES cell-derived renewable and functional midbrain dopaminergic progenitors
10.1073/pnas.1016443108 · 2011 · External reference
Preclinical Analysis of Fetal Human Mesencephalic Neural Progenitor Cell Lines: Characterization and Safety In Vitro and In Vivo
10.5966/sctm.2015-0228 · 2017 · External reference
Embryonic stem cells develop into functional dopaminergic neurons after transplantation in a Parkinson rat model
10.1073/pnas.022438099 · 2002 · External reference
Single cell transcriptomics identifies stem cell-derived graft composition in a model of Parkinson's disease
10.1038/s41467-020-16225-5 · 2020 · External reference
Lineage tracing of stem cell-derived dopamine grafts in a Parkinson's model reveals shared origin of all graft-derived cells
10.1126/sciadv.adn3057 · 2024 · External reference
Patterns of cell death and dopaminergic neuron survival in intrastriatal nigral grafts
10.1006/exnr.1999.7198 · 1999 · External reference
The time course of loss of dopaminergic neurons and the gliotic reaction surrounding grafts of embryonic mesencephalon to the striatum
10.1006/exnr.1996.0141 · 1996 · External reference
Lewy bodies in grafted neurons in subjects with Parkinson's disease suggest host-to-graft disease propagation
10.1038/nm1746 · 2008 · External reference
Transfer of host-derived alpha synuclein to grafted dopaminergic neurons in rat
10.1016/j.nbd.2011.05.001 · 2011 · External reference
Parkin disease: a clinicopathologic entity?
10.1001/jamaneurol.2013.172 · 2013 · External reference
Reduced astrocytic reactivity in human brains and midbrain organoids with PRKN mutations
10.1038/s41531-020-00137-8 · 2020 · External reference
Presence of tau pathology within foetal neural allografts in patients with Huntington's and Parkinson's disease
10.1093/brain/awx255 · 2017 · External reference
A Comprehensive Analysis of Protocols for Deriving Dopaminergic Neurons from Human Pluripotent Stem Cells
10.1002/sctm.18-0088 · 2019 · External reference
Xeno-free induced pluripotent stem cell-derived neural progenitor cells for in vivo applications
10.1186/s12967-022-03610-5 · 2022 · External reference
GMP-grade neural progenitor derivation and differentiation from clinical-grade human embryonic stem cells
10.1186/s13287-020-01915-0 · 2020 · External reference
Clinically compliant cryopreservation of differentiated retinal pigment epithelial cells
10.1016/j.jcyt.2024.01.014 · 2024 · External reference
Determining the optimal stage for cryopreservation of human embryonic stem cell-derived retinal pigment epithelial cells
10.1186/s13287-022-03141-2 · 2022 · External reference
Pluripotent Stem Cell-Based Cell Therapy-Promise and Challenges
10.1016/j.stem.2020.09.014 · 2020 · External reference
Somatic coding mutations in human induced pluripotent stem cells
10.1038/nature09805 · 2011 · External reference
Dynamic changes in the copy number of pluripotency and cell proliferation genes in human ESCs and iPSCs during reprogramming and time in culture
10.1016/j.stem.2010.12.003 · 2011 · External reference
Human pluripotent stem cells recurrently acquire and expand dominant negative P53 mutations
10.1038/nature22312 · 2017 · External reference
Safeguarding genomic integrity in pluripotent stem-cell therapies
10.1016/s0140-6736(26)00095-4 · 2026 · External reference
The consequences of recurrent genetic and epigenetic variants in human pluripotent stem cells
10.1016/j.stem.2022.11.006 · 2022 · External reference
ISSCR standards for the use of human stem cells in basic research
10.1016/j.stemcr.2023.08.003 · 2023 · External reference
Wnt1-lmx1a forms a novel autoregulatory loop and controls midbrain dopaminergic differentiation synergistically with the SHH-FoxA2 pathway
10.1016/j.stem.2009.09.015 · 2009 · External reference
Genetic engineering of mouse embryonic stem cells by Nurr1 enhances differentiation and maturation into dopaminergic neurons
10.1046/j.1460-9568.2002.02255.x · 2002 · External reference
Dopamine neurons derived from embryonic stem cells function in an animal model of Parkinson's disease
10.1038/nature00900 · 2002 · External reference
Engineering synucleinopathy-resistant human dopaminergic neurons by CRISPR-mediated deletion of the SNCA gene
10.1111/ejn.14286 · 2019 · External reference
Host-to-graft propagation of inoculated alpha-synuclein into transplanted human induced pluripotent stem cell-derived midbrain dopaminergic neurons
10.1016/j.reth.2023.12.019 · 2024 · External reference
Regulatory T cells aid stem-cell therapy for Parkinson's disease
10.1038/d41586-023-02177-5 · 2023 · External reference
Trial of Prasinezumab in Early-Stage Parkinson's Disease
10.1056/nejmoa2202867 · 2022 · External reference
Trial of Cinpanemab in Early Parkinson's Disease
10.1056/nejmoa2203395 · 2022 · External reference
An optimized Nurr1 agonist provides disease-modifying effects in Parkinson's disease models
10.1038/s41467-023-39970-9 · 2023 · External reference
