Cell stem cell · 2026
Genomic and transcriptomic quality control for an autologous iPSC-derived cell therapy for Parkinson's disease.
СтатьяНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Bratt-Leal Andres M, Gorba Thorsten, Williams Roy M, Mossman Jim A, Hills Rachel, Tran Ha, Barken Derren, Beauvais Genevieve, Fremgen Daniel, Zhang Ai, Pivaroff Cullen, Lynch Candace, Liu Yanzheng, Chandrasekaran Chandnee, Tastad David, Bridge Charlotte, Wakeman Dustin R, Nguyen Uyen, Chamberlain Brent, Lane Emma L, Lelos Mariah J, Loring Jeanne F, Wirth Edward D, Zhang Xiaokui
Аннотация
Toward development of an autologous, induced pluripotent stem cell (iPSC)-based cell therapy for Parkinson's disease (PD), we demonstrate successful, reproducible genomic and transcriptomic qualification of patient-derived dopaminergic neuron precursor cells (DANPCs) across multiple donors. Our analysis includes whole-genome sequencing data from fibroblasts, iPSCs, and DANPCs and the development of NeuriTest, an RNAseq-based bioinformatic analysis of DANPCs designed to predict cell quality based on empirical animal data. Autologous cell therapies are immune matched to the patient, potentially augmenting durability of benefit compared to allogeneic cells while negating the need for immunosuppression and accompanying side effects. Patient-specific iPSCs are an autologous cell source that can be differentiated to dopaminergic neurons, the cell type lost in PD. We report here our preclinical manufacturing strategy and results demonstrating efficacy in a PD rodent model and safety in a 9-month GLP toxicology study.