Generation of adenosine-kinase-deficient human neuroepthelial stem cells for cell-based delivery of adenosine into the central nervous system.

Daniel Poppe, Jonas Doerr, Julius A Steinbeck, Andreas Reik, David E Paschon, Philip D Gregory, Robrecht Raedt, Paul Boon, Philipp Koch, Oliver Brüstle

Research output: Contribution to conferencePoster

Abstract

As powerful regulator of cellular homeostasis and metabolism, adenosine is
involved in multiple neurological processes including cognition, pain, memory,
depression, schizophrenia or epilepsy. Based on its protective properties,
adenosine has also been considered as therapeutic agent for various brain
disorders. Systemic application of adenosine is hampered by serious side
effects. Recent attempts aim at improving local delivery by depots, pumps or
cell-based applications. Here we report on the generation of adenosine-
releasing hESC-derived neuroepithelial stem cells (lt-NES cells) using zincfinger
nuclease (ZFN)-mediated targeting of the adenosine kinase (ADK) gene. ADK-
deficient lt-NES cells showed a more than 200-fold increase in adenosine
release compared to control cells, which was stable over time and after
differentiation into neurons and glia. We expect these cells to serve as useful
tool for the local therapeutic delivery of adenosine into the central nervous
system.
Original languageEnglish
Pages1
Number of pages1
Publication statusPublished - 22 Nov 2013
EventCell Symposia: Using Stem Cells to Model and Treat Human Disease - Cedars-Sinai Medical Center, Los Angeles, United States
Duration: 21 Nov 201323 Nov 2013
http://www.cell-symposia.com/stemcells-modelingtreatingdisease-2013/

Conference

ConferenceCell Symposia: Using Stem Cells to Model and Treat Human Disease
CountryUnited States
CityLos Angeles
Period21/11/1323/11/13
Internet address

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Poppe, D., Doerr, J., Steinbeck, J. A., Reik, A., Paschon, D. E., Gregory, P. D., ... Brüstle, O. (2013). Generation of adenosine-kinase-deficient human neuroepthelial stem cells for cell-based delivery of adenosine into the central nervous system.. 1. Poster session presented at Cell Symposia: Using Stem Cells to Model and Treat Human Disease, Los Angeles, United States.