Stem Cell Translation Laboratory (SCTL)

National Center For Advancing Translational Sciences

Investigators

Linked publications & trials

Abstract

The progress made in stem cell biology over the past decade has opened up exciting new opportunities for basic and translational scientists worldwide. One recent breakthrough in the field has come from the Stem Cell Translation Laboratory (SCTL), in which we discovered a combination of small molecules (CEPT cocktail) that dramatically improves viability of iPSCs for cryopreservation and single cell cloning. The CEPT cocktail 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. The primary focus of SCTL scientists in the past year has been to develop 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), trophectoderm (e.g., placental development), nociceptors (e.g., opioid research), and astrocytes (e.g., Alexander disease). In FY23, our lab published manuscripts detailing the differentiation of the latter two cell types and a method for efficient and safe single-cell cloning. Additionally, SCTL published a manuscript and launched a free web application (https://sequin.ncats.io/app/) that allows researchers without bioinformatics expertise to easily analyze and visualize single-cell and bulk RNA-seq data and generate publication-ready figures. Ongoing Collaborations: 1) Clifford Woolf (Harvard): Characterization of iPSC-derived nociceptors Collaboration between SCTL and Harvard 2) Shuibing Chen (Cornell University): Multi-omic characterization of type 2 diabetes patient iPSC-derived cell types collaboration between SCTL, NHGRI, Cornell and Columbia 3) Claudia Doege (Columbia University): Multi-omic characterization of type 2 diabetes patient iPSC-derived cell types collaboration between SCTL, NHGRI, Cornell and Columbia 4) Bruce Bean (Harvard University): Characterization of iPSC-derived nociceptors Collaboration between SCTL and Harvard 5) David Bennett (University of Oxford): Transcriptomic analysis of diabetic peripheral neuropathy patient iPSC-derived cell types 6) Ahmet Hoke (Johns Hopkins University): Developing iPSC-derived Satellite Glial Cells for Drug Development 7) Anna Moreno (Navega Therapeutics): Testing a Reversible Gene Editing Method for Analgesia using iPSC-derived Sensory Neurons 8) Rosalind Segal (Harvard University): Studies of Chemotherapy Induced Peripheral Neuropathy (CIPN) in iPSC derived peripheral sensory neurons 9) Martin Schneider (University of Maryland Baltimore): Electrophysiological characterization of iPSC-derived neural cell types 10) Erick Hernandez-Ochoa (University of Maryland Baltimore): Electrophysiological characterization of iPSC-derived neural cell types 11) Laura Pollard (Greenwood Genetic Center): Preclinical development of potential gene editing therapy for Free Sialic Acid Storage Disorder collaboration between SCTL and the FSASD Consortium 12) Richard Steet (Greenwood Genetic Center): Preclinical development of potential gene editing therapy for Free Sialic Acid Storage Disorder collaboration between SCTL and the FSASD Consortium 13) Raymond Wang (Childrens Hospital of Orange County): Preclinical development of potential gene editing therapy for Free Sialic Acid Storage Disorder collaboration between SCTL and the FSASD Consortium 14) Monkol Lek (Yale University): Preclinical development of potential gene editing therapy for Free Sialic Acid Storage Disorder collaboration between SCTL and the FSASD Consortium 15) Kostantin Dobrenis (Albert Einstein College of Medicine): Preclinical development of potential gene editing therapy for Free Sialic Acid Storage Disorder collaboration between SCTL and the FSASD Consortium 16) Steven Walkley (Albert Einstein College of Medicine): Preclinical development of potential gene editing therapy for Free Sialic Acid Storage Disorder collaboration between SCTL and the FSASD Consortium 17) Christine Anne-Longin (University of Paris): Preclinical development of potential gene editing therapy for Free Sialic Acid Storage Disorder collaboration between SCTL and the FSASD Consortium 18) Bruno Gasnier (University of Paris): Preclinical development of potential gene editing therapy for Free Sialic Acid Storage Disorder collaboration between SCTL and the FSASD Consortium 19) Martin Kampmann (UCSF): Characterization and genetic screening of human iPSC-derived astrocytes