Modeling the Genetic Complexity of Parkinson’s Disease: Generation of Patient-Specific iPSCs with Co-occurring SNCA/GBA1 and PRKN/LRRK2 Variants
Objective: This study aimed to generate and characterize human induced pluripotent stem cell (iPSC) lines derived from two Parkinson’s Disease (PD) patients carrying combinations of…Preservation of Cognition Following Intravenous Mesenchymal Stem Cell Therapy in Parkinson’s Disease: Secondary Analysis of a Randomized Trial
Objective: To evaluate longitudinal changes in global cognition following intravenous allogeneic bone marrow–derived mesenchymal stem cell (allo-hBM-MSC) therapy in Parkinson’s disease (PD). Background: Cognitive impairment…Robot-Assisted Transplantation of Hypoimmune iPSC-Derived Dopaminergic Progenitor Cells for PRKN-EOPD
Objective: This study aimed to evaluate the safety and preliminary efficacy of NCR201 transplantation in a PRKN-EOPD patient, providing a first proof-of-concept for this hypoimmune…STEM-PD: An open-label phase I/II trial of transplanted human pluripotent stem cell-derived dopaminergic progenitor cells in Parkinson’s disease
Objective: The STEM-PD trial evaluates safety, tolerability, and feasibility of intraputaminal transplantation of STEM-PD, a dopaminergic(DA) progenitor product derived from a clinical-grade human embryonic stem…Brain-targeted dual-loaded extracellular vesicles enable PROTAC-mediated LRRK2 degradation in Parkinson’s disease
Objective: To develop a brain-targeted extracellular vesicle (EV) platform capable of delivering proteolysis-targeting chimeras (PROTACs) across the BBB and inducing degradation of pathogenic proteins in…Investigating Mitochondrial Integrity in Spinocerebellar Ataxia Type 12 via Patient-Derived iPSC Models
Objective: This study aims to establish patient-derived induced pluripotent stem cell (iPSC) models of Spinocerebellar Ataxia Type 12 (SCA12) and investigate mitochondrial transcriptional alterations across…Restoring Dopaminergic Function: iPSC-derived MSCs Show Therapeutic Promise in Parkinson’s Disease Rat Model
Objective: This study looked into the therapeutic potential of human induced pluripotent stem cell derived mesenchymal stromal cells (iMSCs) in a MPTP induced Parkinson’s disease rat model. Background: PD…Robot-Assisted Transplantation of Hypoimmune iPSC Derived Dopaminergic Progenitor Cells for Idiopathic Parkinson’s Disease
Objective: This study aimed to evaluate the 12-month safety and preliminary efficacy of NCR201 transplantation in idiopathic PD patients, providing a proof-of-concept for universal cell…Developing a novel Disease-Specific iPSC Model for Studying MSA Pathogenesis
Objective: This study aims to develop a human iPSC-derived oligodendrocyte (iPSC-OL) model to investigate cell-specific disease mechanisms in MSA. Specifically, we seek to:a)Generate MSA patient-derived…Intrathecal Administration of Human Oral Mucosa Neural Crest Stem Cells in Moderate/Advanced Multiple System Atrophy: Report on Ongoing Phase 1 Study
Objective: This first-in-human study aims to evaluate the safety of hOMNSC300 following intrathecal administration in patients with moderate to advanced MSA. Background: Stem cell therapy…
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