Scientific American November 2021 ppS2 “Gene Therapy Comes of Age” by Lauren Gravitz
See this special issue of Scientific American for all the details of this editorial and other articles on Gene Therapy
Summary offered by 2244
Image GAO.GOV
“Gene therapy has come a long way since its first human proof-of-concept trials in the 1990s.” Since then there have been ups and some “devastating missteps.”
In the last few years the field has continued to evolve and now includes “not just direct, permanent changes to a cell’s DNA but also transient changes to how genes are translated into proteins.”
Successes have included alleviating “some cases of blindness, cured cancers, addressed the underlying cause of sickle cell disease, and begun to treat congenital disorders, such as spinal muscular atrophy, that might otherwise be lethal.”
Some molecular tools have been critical in recent successes including “a range of new viral vectors” that are effective and safe and the Nobel Prize-winning CRISPR technology.
With these therapies brings hope but the field must, as the next big challenge, control over-expectation, affordability and accessibility.
Comments