Journal of Clinical Investigation
Gene therapy makes mice breath easier
Published in Drug Law Weekly, January 5th, 2010
Individuals with single-gene mutations that mean they have abnormally low levels of the protein alpha-1 antitrypsin are highly susceptible to emphysema, a progressive lung disease that causes severe shortness of breath. Previous attempts to correct single-gene defects that cause lung disease by gene transfer have failed to achieve sustained gene expression in the mouse lung. However, a team of researchers, at Boston University School of Medicine, has now developed an approach that enabled them to attain sustained in vivo expression of normal human alpha-1 antitrypsin at levels able to improve emphysema in mice.
The team, led by Darrell Kotton, introduced gene-carrying...
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Source: Drug Law Weekly (2010-01-05)
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