Induced Pluripotent Stem Cells Generated from Patients with ALS Can Be Differentiated into Motor Neurons

TLDR

Generating patient‑derived pluripotent stem cells could enable large‑scale production of disease‑affected cell types for modeling, drug discovery, and autologous therapies, but it is unclear whether elderly patients with chronic disease can be reprogrammed. We successfully reprogrammed fibroblasts from an 82‑year‑old familial ALS patient into iPS cells that retained embryonic stem cell characteristics and differentiated into motor neurons.

Abstract

The generation of pluripotent stem cells from an individual patient would enable the large-scale production of the cell types affected by that patient's disease. These cells could in turn be used for disease modeling, drug discovery, and eventually autologous cell replacement therapies. Although recent studies have demonstrated the reprogramming of human fibroblasts to a pluripotent state, it remains unclear whether these induced pluripotent stem (iPS) cells can be produced directly from elderly patients with chronic disease. We have generated iPS cells from an 82-year-old woman diagnosed with a familial form of amyotrophic lateral sclerosis (ALS). These patient-specific iPS cells possess properties of embryonic stem cells and were successfully directed to differentiate into motor neurons, the cell type destroyed in ALS.