New therapeutic approaches to mendelian disorders.
@article{Dietz2010NewTA,
title={New therapeutic approaches to mendelian disorders.},
author={H. Dietz},
journal={The New England journal of medicine},
year={2010},
volume={363 9},
pages={
852-63
}
}Thanks to the power of a method to identify etiologic mutations (and hence “causative” genes) in mendelian disease, the molecular mechanisms that give rise to many such diseases are now known. This knowledge has fueled new therapeutic approaches, which are reviewed in this article, the third in the Genomic Medicine series.
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