Many diseases are caused by a missing or defective copy of a single gene. For decades, scientists have been working on gene therapy treatments that could cure such diseases by delivering a new copy of the missing genes to the affected cells.
Despite those efforts, very few gene therapy treatments have been approved by the FDA. One of the challenges to developing these treatments has been achieving control over how much the new gene is expressed in cells — too little and it won’t succeed,…
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News Source: www.sciencedaily.com
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