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FARAFARA Cure FA

Featured Scientist: Mar. 2015

Profile

Dr. David Reid Corey

By David Woods, PhD.

A researcher into genetics with his own enviable genes

If there's any truth in the phrase publish or perish, David Corey is hardly in mortal danger: he has served as the author or co-author of some 121 research publications. Not only that, but he comes to his position as a relatively new FA researcher with an impeccable pedigree: His father, Elias James Corey, won the Nobel Prize for chemistry in 1990… and still serves as a Harvard professor at the age of 86. So David has some pretty good genes both for scientific attainment and for longevity.

David was recently funded by FARA. In his grant proposal, he built upon observations that FA patients have an expanded GAA repeat region within the FXN-1 gene. This expanded repeat leads to reduced production of FXN-1 protein and is the root cause for FA. David and his team have designed small man-made RNAs to recognize the GAA repeat region and guessed that this recognition might block the contacts that lead to reduced production of FXN-1. This hunch paid off––the team discovered that introducing these small RNAs into cells from Friedreich’s patients increased levels of FX-1 protein levels similar to those observed in normal cells. These data suggest that RNAs can be used to up-regulate FXN-1 expression and might provide a new strategy for therapy.

David acquired his PhD in chemistry at the University of California, Berkeley, and is currently a professor in the University of Texas Southwestern Medical Center at Dallas, in the department of pharmacology. He has served on multiple boards and associations… and as a consultant to pharmaceutical companies such as ISIS and Vertex. He is also executive editor of Nucleic Acids Research.

As for the prospect of a cure for FA, David says that ‘while our research is off to a fast start, many hurdles remain before our lab discoveries can be tested in patients. But ‘my lab has been interested in the fundamental aspects of biology and DNA and RNA, both of which code for frataxin. The challenge is how to modify DNA and RNA and how to change gene expression using markers that can recognize DNA and RNA. Our work is attracting increasing support because RNAs that enhance expression of tri-nucleotide repeat genes are a novel concept; and because the mechanism of the RNAs provides insights into the endogenous mechanism of transcriptional silencing of the mutant locus. Further, he believes that finding starting points for treating FA is an important clinical goal with the potential to affect thousands of patients.

Friedreich’s is not the only disease that presents these challenges. David and his team have had a lot of success with protein expression in Huntington's disease, which bears some similarities to FA, he says.

At present two of David's 10 researchers are engaged in work on Friedreich’s ataxia.’ I come into my lab each morning’ he says, trying to achieve things that have never been done before. At 51, David Corey has achieved much. And, as he recognizes, there’s still much more to be done. And he surely has the credentials, the experience and, yes, the genes to do it.

 

About the Author

David Woods

Communications Advisor