IPSC-derived cortical neural progenitor cells

Abstract

This invention provides a regenerative source of transplant material suitable for the treatment of neurodegenerative diseases, and a method for generating cells that can engraft in a transplant host. [Solution] This specification describes the production of neural progenitor cell lines (NPCs) derived from human induced pluripotent stem cells (iPSCs). These iPSC-derived NPCs efficiently engraft in the spinal cord of an ALS animal model, similar to fetal-derived cells used in clinical research, providing neuroprotection to affected motor neurons. Clonal lines were generated using a single-copy GDNF construct inserted into the AAVS1 safe-landing site, including inducible expression of GDNF. These novel iPSC-derived NPC lines are scalable to clinically relevant yields, uniformly produce GDNF, are safe, and present a promising new combination therapy for neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS).

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