Readings

WEEK # TOPICS READINGS
1

Introduction to the Course

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2

Embryonic Stem Cells, Gene Targeting and Transgenic Animals

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Martin, G. R. "Isolation of a Pluripotent Cell Line from Early Mouse Embryos Cultured in Medium Conditioned by Teratocarcinoma Stem Cells." Proceedings of the National Academy of Sciences of the United States of America 78, no. 12 (1981): 7634–8.

Mansour, S. L., K. R. Thomas, et al. "Disruption of the Proto-Oncogene int-2 in Mouse Embryo-Derived Stem Cells: A General Strategy for Targeting Mutations to Non-Selectable Genes." Nature 336 (1988): 348–52.

3

Transgenic Animals in Complex Disease Modeling

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Joshi, R. L., B. Lamothe, et al. Targeted Disruption of the Insulin Receptor Gene in the Mouse Results in Neonatal Lethality. The EMBO Journal 15, no. 7 (1996): 1542–7.

Forlino, A., F. D. Porter, et al. Use of the Cre/lox Recombination System to Develop a Non-lethal Knock-in Murine Model for Osteogenesis Iimperfecta with an α1(I) G349C Substitution. Variability in Phenotype in BrtlIV Mice. The Journal of Biological Chemistry 274 (1999): 37923–31.

4

Regenerative Medicine: Human Embryonic Stem Cells

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Thomson, J. A., J. Itskovitz-Eldor, et al. Embryonic Stem Cell Lines Derived from Human Blastocysts. Science 282, no. 5391 (1998): 1145–7.

Pagliuca, F. W., J. R. Millman, et al. Generation of Functional Human Pancreatic β Cells in Vitro. Cell 159, no. 2 (2014): 428–39.

5

Nuclear Transfer and Cellular Reprogramming

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Elsdale, T. R., Gurdon, J. B., et al. A Description of the Technique for Nuclear Transplantation in Xenopus Laevis. Journal of Embryology and Experimental Morphology 8 (1960): 437–44.

Takahashi, K., and S. Yamanaka. Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors. Cell 126, no. 4 (2006): 663–76.

6

Induced Pluripotent Stem Cells and Disease Modeling

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Hanna, J., M. Wernig, et al. Treatment of Sickle Cell Anemia Mouse Model with iPS Cells Generated from Autologous Skin. Science 318, no. 5858 (2007): 1920–3.

Kondo, T., M. Asai, et al. Modeling Alzheimer's Disease with iPSCs Reveals Stress Phenotypes Associated with Intracellular Aβ and Differential Drug Responsiveness. Cell Stem Cell 12, no. 4 (2013): 487–96.

7

Large Transgenic Mammalian Model Systems

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Li, Z. Y., F. Wong, et al. Rhodopsin Transgenic Pigs as a Model for Human Retinitis Pigmentosa. Investigative Ophthalmology & Visual Science 39, no. 5 (1998): 808–19.

Yang, B., J. Wang, et al. Characterization of Bioactive Recombinant Human Lysozyme Expressed in Milk of Cloned Transgenic Cattle. PloS One 6 (2011): e17593.

8

Oral Presentations

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No Readings
9

Ethical Concerns with Stem Cell Biology

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Sandel, M. J . Embryo Ethics—The Moral Logic of Stem-cell Research. The New England Journal of Medicine 351 (2004): 207–9.

McHugh, P. R. Zygote and "Clonote"—The Ethical Use of Embryonic Stem Cells. The New England Journal of Medicine 351 (2004): 209–11.

10

ZFN and TALEN Strategies for Genome Editing and Disease Modeling

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Chang, C. J., and E. E. Bouhassira. Zinc-finger Nuclease-mediated Correction of α-thalassemia in iPS Cells. Blood 120, no. 19 (2012): 3906–14.

Kato, T., K. Miyata, et al. Production of Sry knockout Mouse Using TALEN Via Oocyte Injection. Scientific Reports 3 (2013): 3136.

11

CRISPR/Cas9 Strategies for Genome Editing and Disease Modeling

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Yang, H., H. Wang, et al. One-step Generation of Mice Carrying Reporter and Conditional Alleles by CRISPR/Cas-mediated Genome Engineering. Cell 154, no. 6 (2013): 1370–9.

Mandal, P. K., L. M. Ferreira, et al. Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells Using CRISPR/Cas9. Cell Stem Cell 15, no. 5 (2014): 643–52.

12

3D Culture Systems

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Nakano, T., S. Ando, et al. Self-formation of Optic Cups and Storable Stratified Neural Retina from Human ESCs. Cell Stem Cell 10, no. 6 (2012): 771–85.

Schwank, G., B. K. Koo, et al. Functional repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients. Cell Stem Cell 13, no. 6 (2013): 653–8.

13

Transdifferentiation

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Choi, J., M. L. Costa, et al. MyoD Converts Primary Dermal Fibroblasts, Chondroblasts, Smooth Muscle, and Retinal Pigmented Epithelial Cells into Striated Mononucleated Myoblasts and Multinucleated Myotubes. Proceedings of the National Academy of Sciences of the United States of America 87, no. 20 (1990): 7988–92.

Ieda, M., J. D. Fu, et al. Direct Reprogramming of Fibroblasts into Functional Cardiomyocytes by Defined Factors. Cell 142, no. 3 (2010): 375–86.

14

Student Topic of Choice

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 No Readings