Introduction and Course Overview

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The discovery of miRNAs as gene regulators

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Lee, R. C., R. L. Feinbaum, et al. "The C. Elegans Heterochronic Gene Lin-4 Encodes Small RNAs with Antisense Complementarity to Lin-14." Cell 75, no. 5 (1993): 843–54.

Reinhart, B. J., F. J. Slack, et al. "The 21-nucleotide Let-7 RNA Regulates Developmental Timing in Caenorhabditis Elegans." Nature 403, no. 6772 (2000): 901–6.


Roles for miRNAs beyond gene downregulation

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Cassidy, J. J., A. R. Jha, et al. "miR-9a Minimizes the Phenotypic Impact of Genomic Diversity by Buffering a Transcription Factor." Cell 155, no. 7 (2013): 1556–67.

Schmiedel, J. M., S. L. Klemm, et al. "Gene Expression MicroRNA Control of Protein Expression Noise." Science 348, no. 6230 (2015): 128–32.


From siRNA discovery to therapeutic agent

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Fire, A., S. Xu, et al. "Potent and Specific Genetic Interference by Double-stranded RNA in Caenorhabditis Elegans." Nature 391, no. 6669 (1998): 806–11.

Querbes, W., R. L. Bogorad, et al. "Treatment of Erythropoietin Deficiency in Mice with Systemically Administered siRNA." Blood 120, no. 9 (2012): 1916–22.


Circular RNAs and the competing endogenous RNA hypothesis

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Memczak, S., M. Jens, et al. "Circular RNAs are a Large Class of Animal RNAs with Regulatory Potency." Nature 495, no. 7441 (2013): 333–38.

Poliseno, L., L. Salmena, et al. "A Coding-independent Function of Gene and Pseudogene mRNAs Regulates Tumour Biology." Nature 465 no. 7301 (2010): 1033–8.


Role of lncRNAs in X chromosome inactivation

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Brown, C. J., B. D. Hendrich, et al. "The Human XIST Gene: Analysis of a 17 kb Inactive X-specific RNA that Contains Conserved Repeats and is Highly Localized within the Nucleus." Cell 71, no. 3 (1992): 527–42.

Zhao, J., B. K. Sun, et al. "Polycomb Proteins Targeted by a Short Repeat RNA to the Mouse X Chromosome." Science 322, no. 5902 (2008): 750–6.


Regulation of genomic imprinting by long non-coding RNAs

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Huang, H. S., J. A. Allen, et al. "Topoisomerase Inhibitors Unsilence the Dormant Allele of Ube3a in Neurons." Nature 481, no. 7380 (2011): 185–9.

Meng, L., R. E. Person, et al. "Ube3a-ATS is an Atypical RNA Polymerase II Transcript that Represses the Paternal Expression of Ube3a." Human Molecular Genetics 21, no. 13 (2012): 3001–12.


Field trip

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Catalytic functions of RNA and the RNA-world hypothesis: Ribozymes

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Cech, T. R., A. J. Zaug, et al. "In Vitro Splicing of the Ribosomal RNA Precursor of Tetrahymena: Involvement of a Guanosine Nucleotides in the Excision of the Intervening Sequence." Cell 27, no. 3 (1981): 487–96.

Sczepanski, J. T., and G. F. Joyce. "A Cross-chiral RNA Polymerase Ribozyme." Nature 515, no. 7527 (2014): 440–2.


It's all about the ribosome

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Noller, H. F., V. Hoffarth, et al. "Unusual Resistance of Peptidyl Transferase to Protein Extraction Procedures." Science 256, no. 5062 (1992): 1416–9.

Kiss-László, Z., Y. Henry, et al. "Site-specific Ribose Methylation of Preribosomal RNA: A Novel Function for Small Nucleolar RNAs." Cell 85, no. 7 (1996): 1077–88.


Chemical modifications of mature RNA molecules

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Wang, X., B. S. Zhao, et al. "N6-methyladenosine Modulates Messenger RNA Translation Efficiency." Cell 161, no. 6 (2015): 1388–99.

Sharma, P. M., M. Bowman, et al. "RNA Editing in the Wilms' Tumor Susceptibility Gene, WT1." Genes Development 8, no. 6 (1994): 720–31.


Harnessing an RNA-guided immune system to treat human disease

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Jinek, M., K. Chylinski, et al. "A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity." Science 337, no. 6096 (2012): 816–21.

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.

Optional Viewing

iBioMagazine. "Jennifer Doudna: Genome Engineering with CRISPR-Cas9." March 23, 2015. YouTube. (16 minutes)


Oral Presentations

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