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Friday, February 5

 

iPS CELLS FOR REGENERATIVE HEALING

8:30 AM Chairperson’s Opening Remarks

Timothy J. Kamp, M.D., Ph.D., Professor of Medicine & Physiology; Co-Director Stem Cell and Regenerative Medicine Center, University of Wisconsin

8:35 microRNA-Target Gene Networks as Fundamental Factors in the Next Generation Regenerative Strategies

Preethi H. Gunaratne, Ph.D., Assistant Professor, Department of Biology & Biochemistry, University of Houston

MicroRNAs are small non-coding RNAs that integrate multiple genes within and across biological pathways. LIN28/let-7; c-MYC-E2F/miR-17-92 and Oct4/Sox2/miR-302-cyclin D1 networks have been tightly linked to embryonic (ES) and more recently to iPS cells. We have also uncovered additional miRNAs regulated by Ronin, a non-canonical pluripotency factor that target genes regulating cytoskeletal remodeling and epigenetic silencing. Discussed is the potential role of these key miRNAs in the next generation regenerative strategies.

9:05 iPS Cells Offer New Approach to Therapy in Thalassemia and Sickle Cell Anemia and Option in Prenatal Diagnosis in Genetic Diseases

Yuet Wai Kan, M.D., Professor, Genetics, University of California, San Francisco

9:35 Pluripotent Stem Cells Derived from Adult Human Testes

Martin Dym, Ph.D., Professor, Biochemistry & Molecular & Cellular Biology, Georgetown University Medical Center

Male germline stem cells obtained from adult human testes can be reprogrammed spontaneously to generate pluripotent stem cells. The production of these “non-canonical” iPS cells is spontaneous, and do not require the addition of exogenous genes, some of which may be cancer causing. Our results suggest that human spermatogonial stem cells have great potential for cell-based, autologous organ regeneration therapy for various diseases and it is thus possible that in the near future men could be cured of disease with a biopsy of their own testes.

10:05 Poster Presentation: Differentiation of Human Embryonic and Human Induced Pluripotent Stem Cells Along the Otic Lineage

Kinuko Masaki, Stanford University

10:20 Coffee Break

 

iPS CELLS FOR REGENERATIVE HEALING CONT’D

11:00 Directed Differentiation of Human iPS Cells Generates Active Motor Neurons

William Lowry, Ph.D., Assistant Professor, Department of Molecular, Cell and Developmental Biology, University of California, Los Angeles

A study of gene expression profiles of mouse and human ESCs and iPSCs suggests that, while iPSCs are quite similar to their embryonic counterparts, a recurrent gene expression signature appears in iPSCs regardless of their origin or the method by which they were generated. Shown is how both hESCs and hiPSCs can differentiate to form fully functional differentiated progeny. We are now setting out to understand whether the differentiated progeny of hESCs and hiPSCs share commonalities or differences as was observed in their undifferentiated parent cells in an attempt to make predictions about whether these two types of pluripotent cells have similar potentials in regenerative medicine.

11:30 Functional Cardiomyocytes Derived from Human Induced Pluripotent Stem Cells

Timothy J. Kamp, M.D., Ph.D., Professor of Medicine & Physiology; Co-Director Stem Cell and Regenerative Medicine Center, University of Wisconsin

Human iPS cells hold great promise for cardiovascular research and therapeutic applications, but the ability of human iPS cells to form functional cardiomyocytes requires careful analysis and optimization.  We provide electrophysiological, pharmacological, and biochemical evidence that iPS cells can differentiate into the three major types of functional cardiomyocytes which can be used in a variety of applications.

12:00 PM Lunch on Your Own

 

iPS CELLS FOR DRUG SCREENING

1:00 Chairperson’s Remarks

 

 

1:05 Featured Speaker

Stem Cells and Drug Discovery: The Beginning of a New Era?

Lee Rubin, Ph.D., Director, Translational Medicine, Harvard Stem Cell Institute

 

 

 

 

 

1:35 CATALYST: The Industrialization of Advanced iPSC Technology for Drug Discovery

Dan Shoemaker, Chief Technology Officer, Fate Therapeutics

CATALYST is designed to accelerate the innovation of induced pluripotent stem cell (iPSC) technology in collaboration with industry to support launching a fully-enabled platform in this new paradigm of drug discovery and development. CATALYST is exploring the creation of iPSC-derived, disease-specific model systems that improve the recapitulation of human physiology and more effectively predict clinical response. CATALYST is committed to developing an iPS cell technology platform to accelerate candidate identification and lead validation for drug discovery and development for pharmaceutical members. This session will discuss:

  • The critical elements of industrialization, including cell sourcing, reprogramming, differentiation and commercial supply
  • Approaches for creating non-genetically modified iPS cells and mature phenotypes
  • Quantitative methods to analyze cell states for high-quality differentiation and disease modeling
  • Uses of iPSC technology in drug discovery

Sponsored by

1:50 Sponsored Presentation 

Targeting Muscular Dystrophy: How do we Mimic the In Vivo System?

Lorena Griparic, Ph.D., Research Scientist, DV Biologics

Muscular dystrophy (MD) is a well characterized neuromuscular disorder. Here we show that using cells isolated from different tissues of MD patients and their pedigree is an effective tool for understanding how to treat the disease. Our MD pedigree system is the first commercially available tool allowing the study of this disease and the production of iPS cells.

Sponsored by

2:05 iPSC-enabled Drug Discovery: A Paradigm Shift to Increase POS in the Clinic

Berta Strulovici, Chief Technology Officer, iPierian

Until recently, human disease specific pluripotent stem cells could be made only by tedious genetic modification of existing hES cells or by generating such cells from embryos with diagnosed monogenic diseases. Recent advances using induced pluripotent stem cells (iPSCs) have enabled the production of unlimited numbers of cells with a very specific genetic background that can be used as models for drug discovery. Coupled with the ability of these cells to be differentiated to virtually “any type of cell in the body”, the iPSC technology has the ability to revolutionize the way drug discovery is done today. In my presentation, I will describe the use of human iPSC-based assays for drug discovery in our therapeutic areas of focus.

Sponsored by 

2:20 iPS Cells Panel of Experts

iPS cells have invigorated and united the stem cell research community and strides continue in efficient re-programming.  This is evident through funding and companies investing their future through this revolutionary technology.  Hear the experts in the iPS Cells field as they present their latest technology followed by an interactive panel discussion.

3:05 Close of Conference

 

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