Gene Therapy Is Coming of Age

Tue, 19 Oct 2021 11:30:00 GMT
Scientific American - Technology

Various approaches are approved for treating blood cancers and a few rare disorders—they may soon...

Gene therapy has come a long way since its first human proof-of-concept trials in the 1990s.

The approach-which involves fixing or replacing a disease-causing gene or changing its activity-has recorded some remarkable successes and some devastating missteps.

In the past decade those extreme ups and downs have leveled off, and now gene therapy, in a variety of forms, has begun advancing at a rapid pace.

Over the past few years not only has the discipline changed but the very definition of gene therapy has evolved.

Today the field includes not just direct, permanent changes to a cell's DNA but also transient changes to how genes are translated into proteins.

The history of gene therapy has had a lot to overcome, both in reputation and at the lab bench.

Early tragedies led to a scientific reckoning of sorts-while many researchers turned away from the field entirely, others began to pursue ways to prevent some of the most serious side effects.

What has resulted is a range of new viral vectors, engineered to incorporate their genetic cargo more safely and efficiently into the genome, as well as the rapid adoption and development of other tools, such as the Nobel Prize-winning technique known as CRISPR. Gene therapy has more than compensated for its shaky start, and the field managed to rehabilitate not only its science but its reputation.

Current gene therapy approaches are pricey and not easily available, both issues that limit their possible reach.

Solving these problems may be the field's next big challenge.

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