In 1970, Feidman and Roblin published a seminal paper in Science proposing that genetic diseases could be cured by using 'good' DNA to replace the 'defective' DNA causing genetic disease. This idea - the central tenet of gene therapy - remains sound but has proved unexpectedly difficult to turn into a mainstream therapeutic reality. As of today, the FDA has approved only two gene therapies (as well as one oncolytic virus and 15 cell therapies; eight of which are cord blood suppliers, and four are CAR-T or other cancer immunotherapies).
Here, we look back over the challenges the gene therapy industry has faced from viral vector selection through vector engineering to scaling up manufacture and obtaining regulatory approval.
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