Point mutations represent the majority of known human genetic variants associated with disease but are difficult to correct cleanly and efficiently using standard genome-editing methods. For his lecture, Dr. Liu will describe the development, application, and evolution of base editing, a novel approach to genome editing that directly converts a target base pair to another base pair in living cells without requiring DNA backbone cleavage or donor DNA templates.
Salamanders and starfish might be “simpler” than humans, but they far surpass us in one major way—the ability to regenerate tissues and regrow lost limbs. Dr. Sánchez Alvarado studies regeneration using the flatworm planaria Schmidtea mediterranea. Remarkably, when halved or quartered this organism can clone itself from the pieces. More than 100 years ago, that feat captured the attention of geneticist Thomas Hunt Morgan, who studied planarians years before his famed work on fruit flies. As astonishing as the planarian regenerative capacities are, Dr.
Dr. Young’s laboratory has identified genes that control the circadian rhythms of Drosophila melanogaster. Interactions among these genes and their proteins set up a network of oscillations within single cells. These oscillations are autonomously generated, are found in most tissues, and establish rhythms in physiology and behavior. This mechanism is conserved within the animal kingdom: similar clock genes regulate patterns of sleep and other rhythms in humans.
Genomics has undergone a resolution revolution, such that the nucleic acid content of individual cells are now sequenced routinely in highthroughput mode. Thus we can define the cellular composition of tissues in an unbiased and comprehensive way using single-cell transcriptomics, revealing the molecular fingerprint of cell states and their predicted signaling circuits in tissues across development and disease. Dr. Teichmann will illustrate this with the maternal-fetal interface in the first trimester placenta and decidua.
Dr. Gahl studies the natural history, diagnosis, and treatment of rare genetic disorders such as cystinosis, Hermansky-Pudlak Syndrome, sialic acid storage diseases, GNE myopathy, and disorders of platelets and pigmentation. He also investigates undiagnosed disorders under the aegis of the NIH Undiagnosed Diseases Program and Network, and pursues new disease discovery.
The major interest of Dr. Hotamisligil's laboratory is to study the regulatory pathways, which control glucose and lipid metabolism. His lab's biochemical and genetic studies focus on signal transduction using cultured mammalian cells as well as transgenic animals to identify specific abnormalities in these pathways, which are involved in human metabolic and inflammatory diseases including obesity, diabetes, fatty liver disease, atherosclerosis, and asthma.
While inbred mice have been a very powerful model for analyzing the immune system, recent advances, both technological and conceptual, have begun to make direct studies of the human immune system possible. This is vitally important from a translational perspective, as mouse models of disease have not been as productive as hoped for in producing “actionable intelligence” with which to diagnose and treat patients.
In addition to serving as director of NCI, Dr. Sharpless continues his research in understanding the biology of the aging process that promotes the conversion of normal self-renewing cells into dysfunctional cancer cells. Dr. Sharpless has made seminal contributions to the understanding of the relationship between aging and cancer, and in the preclinical development of novel therapeutics for melanoma, lung cancer, and breast cancer.
Professor Collinge leads a highly multidisciplinary research unit dedicated to understanding prion diseases. Prions are notorious “protein-only” infectious agents devoid of genes which cause invariably fatal brain diseases following silent incubation periods which may span a human lifetime. The diseases can arise spontaneously, by infection or be inherited. Remarkably, prions are composed of a cloud of self-propagating assemblies of a misfolded cellular protein that can encode information, generate neurotoxicity and evolve and adapt in vivo.
The research in Cory Abate-Shen’s laboratory is focused on understanding basic mechanisms of transcriptional regulation and differentiation, and how these become dysregulated in cancer. The laboratory takes a multi-disciplinary approach to investigate genitourinary malignancies, which includes using mechanism-based studies, analyses of genetically-engineered mouse models (GEMMs), and state-of-the-art systems biology approaches.
The page was last updated on Thursday, September 27, 2018 - 2:19pm