Stem Cell Translation Laboratory (SCTL)

National Center For Advancing Translational Sciences

Investigators

Linked publications, trials & patents

Abstract

The progress made in stem cell biology over the past decade has opened up exciting new opportunities for basic and translational scientists worldwide. Induced pluripotent stem cells (iPSCs) are particularly useful because scientists can differentiate them into many different cell types that are relevant for drug discovery and clinical therapies. Stem Cell Translation Laboratory (SCTL) scientists investigate iPSCs that have been derived by reprogramming adult cells (e.g., skin cells) into embryonic-like stem cells. These patient and disease-specific cells have the potential to become any cell type of the human body. SCTL scientists are developing the most rigorous and efficient iPSC differentiation protocols for a variety of cell types relevant for both disease modeling and cell therapy applications, including dopamine neurons (e.g., Parkinsons disease), motor neurons (e.g., Lou Gehrigs disease), insulin-producing cells (e.g., type 1 diabetes), hepatocytes (e.g., liver failure), nociceptors (e.g., opioid research), astrocytes (e.g., Alexander disease), trophectoderm (e.g., placental development), and more. SCTL is also using various iPSC-derived cell types as models for studying SARS-CoV-2 infection and drug discovery. We are also developing new quality control measures for quality control, genome stability, and cell maturation. One recent breakthrough is the discovery that a combination of small molecules, the so-called CEPT cocktail, dramatically improves viability of iPSCs for cryopreservation and single cell cloning. The CEPT cocktail now allows for efficient and streamlined genome editing with CRISPR/Cas in pluripotent stem cell lines as well as high-throughput robotic biomanufacturing, among other applications. Ongoing Collaborations: 1) Ravi Iyengar, Eric Sobie, Christoph Schaniel (Mount Sinai): A library of signatures from iPSCs and their derived cardiomyocytes and neurons collaboration between SCTL and LINCS Consortium 2) Mustafa Sahin (Childrens Hospital, Harvard Medical School): Optimization of iPSC-derived GABAergic neuron differentiation protocols 3) Mark Schurdak (University of Pittsburgh): Cell differentiation protocol for medium-sized spiny neurons for Huntingtons 4) Clive Svendsen, Dhruv Sareen (Cedars-Sinai): A library of signatures from iPSCs and their derived cardiomyocytes and neurons collaboration between SCTL and LINCS Consortium 5) Joseph Wu (Stanford University): TBX5Clover2/NKX2.5TagRFP hiPSCs for isolating human lineage-specific cardiovascular cells 6) Clifford Woolf (Harvard): Characterization of iPSC-derived nociceptors 7) Martin Kampmann (UCSF): Characterization and genetic screening of human iPSC-derived astrocytes