CORTICAL NEURAL PROGENITOR CELLS FROM IPSCS

Described herein is the production neural progenitor cell lines (NPCs) derived from human induced pluripotent stem cells (iPSCs). These iPSC-derived NPCs engraft efficiently into the spinal cord of ALS animal models and provide neuroprotection to diseased motor neurons, similar to the fetal-derived...

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Bibliographische Detailangaben
Hauptverfasser:	SVENDSEN, Clive N, LAPERLE, Alexander, FULTON, Aaron
Format:	Patent
Sprache:	eng ; fre ; ger
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING ORGANIC CHEMISTRY PEPTIDES PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Described herein is the production neural progenitor cell lines (NPCs) derived from human induced pluripotent stem cells (iPSCs). These iPSC-derived NPCs engraft efficiently into the spinal cord of ALS animal models and provide neuroprotection to diseased motor neurons, similar to the fetal-derived cells used in clinical study. Clonal lines were generated with a single copy GDNF construct inserted in the AAVS1 safe landing site, including inducible expression of GDNF expression. These new iPSC-derived NPC lines are scalable to clinically relevant production volumes, uniformly produce GDNF, are safe, and represent a promising new combination therapy for neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS).