The SKI Stem Cell Research Facility provides a number of services to the stem cell community including:

• Teach basic culture of pluripotent stem cells
• Maintain a library of pluripotent stem cells
• Reprogram somatic cells into induced pluripotent stem cells
• Provide genetic modifications
• Directed differentiation of pluripotent stem cells
• Transition protocols to clinical GMP grade processes

We have taught over 120 scientists from more than 40 laboratories. Most take our
Basics in Human Pluripotent Stem Cell Culture class but we also offer advanced and intensive classes in Neural Induction and Bacterial Artificial Chromosome Transgenesis. Our library of human pluripotent stem cells includes the original Wisconsin lines H1, H7, H9, H13 and H14, the Rockefeller lines RU01 and RU02 (kindly provided by Ali Brivanlou), the BioTime lines ESI-017 and ESI-035 and the newer Wisconsin lines WA25-27. We have made hundreds of hiPSC lines in our lab using Lenti- and Sendai-based vectors (Papapetrou et al. 2009; Chambers et al. 2009; Lee et al. 2009; Miller et al. 2013).

In collaboration with Lorenz Studer’s lab, our facility was the first to publish the use of bacterial artificial chromosomes (BACs) in human pluripotent stem cells for transgenics (Placantonakis et al. 2009). We recently extended these findings to use BACs to probe gene function through increases in a gene’s copy number (Harel et al. 2012). Our facility was among the first to demonstrate zinc finger nuclease-enhanced homologous recombination and translocation in a collaboration with Maria Jasin’s lab (Brunet et al. 2009) and later Erika Brunet’s lab (Piganeau et al. 2013).

Our Facility is also part of a
New York State (NYSTEM)-funded consortium that has manufactured a cellular therapy for Parkinson’s disease (MSK-DA01). Our role was to develop the process to be clinically compatible, developing and validating quality control assays, and developing methods for the cryopreservation of midbrain dopamine neurons derived from human embryonic stem cells. We moved our validated process into the Cell Therapy and Engineering Facility led by Isabelle Rivière for large-scale manufacture and successfully manufactured 4 large batches of cells. One will be subjected to a battery of safety tests before a human trial that we hope will take place in 2018.

We hope to share our knowledge and GMP-manufacturing capabilities with the New York State stem cell community this year, thanks to funding from
NYSTEM. We also receive generous funding from The Starr Foundation through the Tri-Institutional Stem Cell Initiative.

Mark J. Tomishima
tomishim@mskcc.org