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Using RNA instead of DNA could avoid the health risks–and the political pitfalls–of stem-cell treatments.
Regulatory approval of a gene therapy treatment in Europe could spark broader patient access to the technology.
Are we prepared to know the genetic flaws of the unborn?
Engineering a patient’s own immune cells to resist HIV could eliminate the need for lifelong antiretroviral therapies.
A Boston startup wants to create precise genome-editing treatments that can address more types of disease than previous gene therapy methods.
Several gene therapies are or will soon be in late-stage human trials. One of them could be the first to get FDA approval for sale in the U.S.
By delivering gene therapies to patients before they go blind, doctors may be able to prevent the loss of many important light-detecting cells.
A compound that helps cells produce normal proteins from wonky genes could have a broad impact on genetic diseases.
In 2012, genomics tiptoed into the doctor’s office, gene therapy rose again, and man and machine united.