Cell Therapies for Parkinson’s Disease

2019 - Clinical and Translational Science - 12 (2): 95-97
Stefan Irion
Irion presents a summary of Parkinson’s disease and cell therapeutic approaches. Read More »

Human Embryonic Stem Cell-Derived Oligodendrocyte Progenitors Remyelinate the Brain and Rescue Behavioral Deficits following Radiation

2015 - Cell Stem Cell - 16 (2): 198-210
Piao, J., Major, T., Auyeung, G., Policarpio, E., Menon, J., Droms, L., Gutin, P., Uryu, K., Tchieu, J., Soulet, D., Tabar, V.
Plao et al illustrate that pluripotent stem cells can be used to derive glial cells (oligodendrocytes) which show significant response benefit in pre-clinical models of radiation brain injury. Read More »

Dopamine neurons derived from human ES cells efficiently engraft in animal models of Parkinson’s disease

2011 - Nature Letter - 480: 547-551
Kriks, S., Shim, J., Piao, J., Ganat, Y.M., Wakeman, D.R., Xie, Z., Carrillo-Reid, L., Auyeung, G., Antonacci, C., Buch, A., Yang, L., Beal, M.F., Surmeier, D.J., Kordower, J.H., Tabar, V., Studer, L.
Kriks et al document pre-clinical model experience in Parkinson’s disease, establishing the foundation for translation. Read More »

Deriving Human ENS Lineages for Cell Therapy and Drug Discovery in Hirschspring’s Disease

2016 - Nature - 431 (7592): 105-109
Fattahi, F., Steinbeck, J.A., Kriks, S., Tchieu, J., Zimmer, B., Kishinevsky, S., Zeltner, N., Mica, Y., El-Nachef, W., Zhao, H., de Stanchina, E., Gershon, M.D., Grikscheit, T.C., Chen, S., Studer, L.
Fattahi et al demonstrate that diseases of the peripheral nervous system be approached using pluripotent stem cells. Ex vivo expansion of enteric neurons and transplantation into mouse models show recovery benefit in models of Hirschsprung’s disease with implications for treatment of diabetic gastroparesis. Read More »

Directed Differentiation of Human Pluripotent Stem Cells to Microglia

2017 - Stem Cell Reports - 8 (6): 1516-1524
Douvaras, P., Sun, B., Wang, M., Kruglikov, I., Lallos, G., Zimmer, M., Terrenoire, C., Zhang, B., Gandy, S., Schadt, E., Freytes, D.O., Noggle, S., Fossati, V.
Douvaras et al describe the derivation of microglia from pluripotent stem cells. Microglia are the fulcrum of immune modulation in the brain and control of this cell population opens new doors for treatment of neuroinflammatory disease. Read More »


Genetically Engineered Macrophages: A Potential Platform for Cancer Immunotherapy

2017 - Human Gene Therapy - 28 (2)
Moyes, K.W., Lieberman, N.A.P., Kreuser, S.A., Chinn, H., Winter, C., Deutsch, G., Hoglund, V., Watson, R., Crane, C.A.
Moyes et al validate approaches to engineer adult macrophages for payload delivery, establishing a platform with widespread utility. This is an effective proof point for functional derivation of similar macrophages derived from pluripotent stem cell sources. Read More »

HLA-E-expressing pluripotent stem cells escape allogeneic responses and lysis by NK cells

2017 - Nature Biotechnology - 35 (8): 765-772
Gornaluss, G.G., Hirata, R.K., Funk, S.E., Riolobos, L., Lopes, V.S., Manske, G., Prunkard, D., Golunga, A.G., Hanafi, L., Clegg, D.O., Turtle, C., Russell, D.W.
Gornalusse et al demonstrate that master cell banks of pluripotent stem cells can be gene edited to ablate HLA receptors and avoid T cell recognition. In addition, these banks are engineered to express a recombinant receptor to prevent innate cell clearance. This escape from immune surveillance is demonstrated using in vitro assays and validated in pre-clinical models. Read More »

Umbilical cord blood–derived T regulatory cells to prevent GVHD: kinetics, toxicity profile, and clinical effect

2019 - Blood - 127: 1044-1051
Brunstein, C.G., Miller, J.S., McKenna, D.H., Hippen, K.L., DeFor, T.E., Sumstad, D., Curtsinger, J., Verneris, M.R., MacMillan, M.L., Levine, B.L., Riley, J.L., June, C.H., Le, C., Weisdorf, D.J., McGlave, P.B., Blazar, B.R., Wagner, J.E.
Brunstein et al describe successful use of cord blood derived regulatory T cells in prevention of graft vs host disease (GvHD). This establishes a clinical response to target in deriving similar cells from pluripotent stem cells. Read More »


Human embryonic stem cell–derived cardiomyocytes restore function in infarcted hearts of non-human primates

2019 - Nature Biotechnology - 36 (9): 899
Liu, Y., Chen, B., Yan, X., Fugate, J.A., Kalucki, F.A., Futakuchi-Tsuchida, A., Couture, L., Vogel, K.W., Astley, C.A., Baldessari, A., Ogle, J., Don, C.W., Steinberg, Z.L., Seslar, S.P., Tuck, Tsuchida, H., Naumova, A.V., Dupras, S.K., Lyu, M.S., Lee, J., Hailey, D.W., Reinecke, H., Pabon, L., Fryer, B.H., MacLellan, W.R., Thies, R.S., Murry, C.E.
Liu et al use a non-human primate pre-clinical study to reach proof of concept for adoptively transferred cardiomyocytes to reconstitute heart muscle and restore failing hearts to normal function. Read More »

Human Pluripotent Stem Cell-Derived Atrial and Ventricular Cardiomyocytes Develop from Distinct Mesoderm Populations

2017 - Cell Stem Cell - 21 (2): 179-194
Lee, J.H., Protze, S.I., Laksman, Z., Backx, P.H., Keller, G.M.
Lee et al demonstrate the recapitulation of cardiomyocyte developmental pathways in producing and characterizing PSC derived cardiomyocytes and their authenticity when compared to adult tissue. Read More »

Human Pluripotent Stem Cells: Prospects and Challenges as a Source of Cardiomyocytes for In Vitro Modeling and Cell-Based Cardiac Repair

2017 - Advanced Drug Delivery Review - 96: 3-17
Hartman, M., Dai, D., Laflamme, M.
Hartman and Laflamme provide a perspective on these studies and describe studies to directly reconstitute cardiomyocytes by ex vivo expansion from pluripotent stem cells. Read More »