Researchers at the University of Iowa have created a zebrafish model of CEP290 LCA, and have been able to restore vision in these embryos by gene therapy! What's more, they showed that only a portion of the gene is enough to suppress the visual impairment. This is of the highest importance, because CEP290 is extremely large, and generating a vector capable of packaging such a large gene has been a challenge. The study by Baye et al, just published in Human Molecular Genetics, has thus shed light on the function of CEP290 specific regions.
The zebrafish is being widely used for research into vertebrate development and gene function. It offers several advantages to researchers, including a fully sequenced genetic code, well-understood and easily observable and testable developmental behaviors, availability of well-characterized mutants and rapid embryonic development.
The full article describing these crucial discoveries can be found online at
http://hmg.oxfordjournals.org/content/early/2011/01/21/hmg.ddr025.abstract
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2 comments:
I just wanted to post and say that I enjoy this blog. My daughter has LCA (RDH12 gene affected). We are excited to be presenting a check to start research on RDH12 to Dr. Bennett in Philadelphia next week! ($70,000)!!! It is great to see that many of the LCA genes are coming alone toward a cure.
www.rdh12.org
www.pletcher5journey.blogspot.com
Thanks for the nice words,and of course for the huge amount of money for LCA research!!! Though each gene is different, they share common traits, and research on one gene may help cure other types of LCA as well.
I think you're on the LCA list, correct?
Cheers
Fran
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