For many years, scientists have been working on gene therapy treatments that could cure diseases caused by a single missing or defective gene. Few therapies have been approved by the US Food and Drug Administration, one impediment being poor control over efficacy and side-effect profile, caused by too much or too little gene being delivered.
Now, researchers at Massachusetts Institute of Technology in Cambridge, have developed a method using a molecular control circuit called an 'incoherent feedforward loop', published in Cell Systems.
The loop allows much finer control of gene therapy and offers hope for those affected by genetic conditions such as Fragile X syndrome.
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