How does simple cell biology donate to biomedicine? A fresh group of Features in offers a cross portion of compelling types of how simple cell biology results can result in therapeutics. fundamental systems of cholesterol receptor trafficking and its own relevance to cardiovascular disease, thanks partly to research on cells from sufferers with familial hypercholesterolemia (Dark brown and Goldstein, 1986). For many years, their collaborative function has served being a shining example of the power of fundamental cell biology in a highly relevant disease establishing. Today, cell biology is definitely a major driver of all aspects of biomedicine. The analysis of a disease progressively relies on genetic, molecular, and cellular markers, and drug discovery offers shifted from blind screening to targeted molecular design knowledgeable by our genetic, molecular, and cellular understanding of a disease. Technological improvements in areas such as purchase PD 0332991 HCl genome sequencing and high-throughput screening have made it possible to visit from a basic finding in the laboratory to a medical trial at an unprecedented pace. As a result, the application of basic research to the medical center can easily happen during the career of a researcher and is a much more attainable goal than in decades past. To highlight the importance of basic cell biology to biomedicine, is publishing a series purchase PD 0332991 HCl of Feature articles, each of which represents a story, often a personal account by a key researcher, to give both historical and practical insight into how basic cell biological observations purchase PD 0332991 HCl have contributed to therapeutic advances. These articles will be freely available online from the time of publication and distributed as a collection in em JCB /em s Special Issue 2013 at the American Society for Cell Biology annual meeting later this year. We begin the series with three articles in this issue of em JCB /em . The first, from Francis Collins and colleagues (Gordon et al., 2012), shows how a chance encounter at a social event led to the surprising discovery that the premature aging syndrome progeria is caused by a mutation in lamin A and led to medical tests in record period. The next (Davis and Schlessinger, 2012) identifies the culmination of years of concentrated, methodical work learning signaling pathways and oncogenic mutations that led Yossi Schlessinger and co-workers to build up a drug to focus on the B-Raf kinase in melanoma. In the 3rd, Frank Bennett and co-workers (Rigo et al., 2012) illustrate how merging the advantages of market and academia can be leading to the introduction of equipment that repair the pre-mRNA splicing defect in vertebral muscular atrophy. In upcoming content articles, Larry Steinman will describe the way the advancement of the powerful multiple sclerosis medication Natalizumab arose from research on receptors involved with immune system cell migration. Gergely Lukacs and co-workers will display how examining the cell biology of disease mutations in the cystic fibrosis transduction route (CFTR) can be informing the introduction of particular reagents to improve route dysfunction. Christine Seidman and co-workers will show insights into cardiac cell biology which have surfaced purchase PD 0332991 HCl from studying pet models of cardiovascular disease. Additional content articles from Larry Goldstein, Johan Auwerx, and Sakari Kauppinen will touch upon a few of the hottest fields in current translational researchstem cells, metabolism, and microRNAsto provide an update on progress in these areas. Not all of the articles are simple success stories; some highlight the difficulties in the path from basic cell biology to the clinic. Oliver Hantschel and Giulio Superti-Furga will present some of the stumbling blocks inherent to the current way that drugs are developed and provide some ideas for methods to streamline the procedure. Fred Goldberg shall graph the introduction of the proteasome inhibitor Bortezomib, an effective treatment for multiple myeloma that experienced major obstacles on the path to the center. Tony Oro will discuss medication level of resistance in basal cell carcinoma and attempts to Rabbit Polyclonal to USP13 comprehend and conquer this growing problem. This piece, like several others, makes the important point that studying the cell biology of disease is not a one-way street; disease cells are extraordinarily powerful, naturally occurring experimental tools that provide novel insight into basic cell biology. Therapeutic discoveries can arise both from the study of an purchase PD 0332991 HCl apparently inconspicuous cell biological pathway that suddenly is found to be at the center of a disease and from the use of disease mutations and animal models of disease to support and inform a basic cell biology research program. Either way, cell biologists are well situated to contribute to understanding the basis of disease and to developing therapeutics. This is a win-win situation for analysts, clinicians, rather than least of all sociable people whose lives are improved by our attempts. We wish how the good examples assembled with this series shall inspire cell biologists.