drug discovery
Cool Videos: Diving for Drugs
Posted on by Dr. Francis Collins
Who says biomedical scientists always have to work indoors? The next installment in our mini-film fest proves otherwise, offering a close-up look at some medicinal chemists who are busy carrying out their research in warm waters off the Florida Keys.
This aquatic adventure may not be as action-packed as “Pirates of the Caribbean” or “Finding Nemo.” But these researchers from the University of Florida College of Pharmacy in Gainesville are out to discover something far more valuable to patients than sunken treasure: marine life with chemical compounds that may provide the basis for new treatments and cures.
Fishing for Answers in Human Disease
Posted on by Dr. Francis Collins
Caption: Researcher Zhaoxia Sun, at Yale, uses the zebrafish to study Polycystic Kidney Disease, which affects more than 600,000 Americans. Mutations in the zebrafish vhnf1 gene, and its human counterpart, cause cysts in both zebrafish and human kidneys (as shown by the large “bubble” seen in the mutant fish). [3]
Credit: Zhoaxia Sun, Biological & Biomedical Sciences, Yale University
Just last month, an international team of scientists—funded in part by NIH—published the entire genetic code of the zebrafish [1]. This is a vital resource for understanding human health and disease. How does the genetic blueprint of a fish help us or accelerate drug discovery? Well, it turns out that more than 75% of the genes that have been implicated in human diseases have counterparts in the zebrafish. So, if we discover a mutation in a human, we can make the corresponding mutation in the zebrafish gene—and often get a pretty good idea of how the gene works, how the mutation causes havoc, and how it causes disease in humans. We can even use the zebrafish to test potential drug candidates, to see whether they can alter or fix the symptoms before moving on to mice or humans.
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