International Stem Cell Registry

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1 International Stem Cell Registry

2 Importance of Stem Cells Stem cells are model systems for the study of development and disease. Pluripotent stem cells offer new tools for drug design and discovery. Pluripotent cells have potential uses in cell replacement therapies.

3 Stem Cells: an evolving field Biology Characteristics Differentiation Technology New methods for deriving pluripotent cells such as ips cells New techniques for growth and differentiation Applications Cell replacement therapy Drug screening Disease models

4 Center for Stem Cell Biology and Regenerative Medicine Key component of Governor Deval Patrick s Massachusetts Life Sciences Initiative Established, and operated with funding from the Massachusetts Life Sciences Center A cornerstone of University of Massachusetts Medical School s commitment to clinical and translational research

5 Center for Stem Cell Biology and Regenerative Medicine Stem Cell Registry Stem Cell Bank Education and Training Research

6 MHSCB Mission: The Massachusetts Human Stem Cell Bank provides human ES and reprogrammed (ips) cell lines to facilitate studies into the properties and applications of pluripotent stem cells. These cells are available to: o academic researchers o commercial operations o the international biomedical research community. The bank provides growth, characterization and distribution of hesc and ips lines, as well as training in pluripotent stem cell culture and differentiation techniques.

7 Training and Education Mass Life Sciences Grant provides funding for training and education for the Stem Cell Center Programs include: K 12 Education for students and teachers Collaboration with local colleges (Middlesex, QCC, WPI) Workshops for the public Technical Training within the stem cell community

8 The need for a comprehensive registry There are hundreds of hes and ips cell lines worldwide, most of which are not published. Pluripotent stem cells have a number of unique characteristics that are used to assess their undifferentiated state and capacity to form different cell types. Different states and funding agencies have different guidelines regarding hes cell line derivation (provenance), which has created a regulatory maze. Published research is not searchable by cell line. Scientists need up to date information to stay current with this rapidly advancing field.

9 International Stem Cell Registry A publicly accessible, searchable, comprehensive and continuously updated database that includes published and validated unpublished information on all pluripotent stem cell lines. Dedicated to acquiring, configuring and disseminating, information between providers, researchers and the broader scientific community. Includes cell lines developed by the international research community.

10 Origins of cell lines represented in the ISCR

11 Registry information Practical Information Developer Provider(s) Provenance Scientific Information Derivation Characterization Marker expression testing HLA and genotyping Literature references

12 International Stem Cell Registry

13 Data Collection Published literature (derivation, characterization, uses) Contact with cell line developers (unpublished data) Websites posting independently derived characterization information (ISCI Stem Cell Forum) Information derived by stem cell banks (QC data, etc.)

14 Data Collection Publications Tools: PubMed only allows search of titles, abstracts an keywords Crossref Not all journals represented, difficult to sort by publication date Publication websites

15 Challenges Many lines are unpublished, making identification, data gathering and validation difficult. Investigators are often unwilling to post unpublished data Privacy issues impede obtaining provenance information. Conflicting information from different sources (lack of standards) Data presentation (creating a user friendly display of complex information) Accelerating pace of research (maintaining up to date information)

16 Coming Attractions Summary Tab for each stem cell profile o NIH Approval o Availability & provider link o Gender & Karyotype o Limited markers o Feeder cells used in derivation o Number of publications in database Advanced Search o Directed differentiation by cell type o Transgenic cell lines o Expanded set of markers o Adherence to specific guidelines Searchable forum for methodologies and other discussions Repository for unpublished & raw data, negative results Modifications as suggested by site users

17 International Stem Cell Registry Curators: Mai X. Luong, PhD Kelly P. Smith, PhD

18

19 Embryonic Stem Cells

20 Embryonic Stem Cells Derivation Isolated from early stage embryos Characteristics Competent to form ALL specialized cells, tissues, and organs of the adult organism (pluripotent) Indefinite life span in culture Limitations Derivation normally results in embryo destruction Prior to March 9, 2009, only a limited number of lines were available for federally funded research Risk of tumors (teratomas) from transplanting undifferentiated cells

21 Reprogrammed cells - Induced Pluripotent Stem (ips) cells

22 Reprogrammed cells Induced Pluripotent Stem (ips) cells Derivation Isolate adult somatic cells (e.g. skin fibroblasts) Viral introduction of genes expressing factors that control very early development Characteristics Re establish pluripotency, and indefinite lifetime in culture. Cells can be developed that are matched to patients. Limitations Must establish functional equivalency of IPS and human embryonic stem cells. Viruses used in derivation may promote tumorigenesis.

outline What is a stem cell? Tissue stem cell Embryonic stem cell ips cell -production -advantages Applications Issue Conclusion

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