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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.
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.
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.
Gene therapy to make cells sensitive to light takes a step toward clinical use.
Are we prepared to know the genetic flaws of the unborn?
New partnerships could help bring a novel class of biopharmaceutical to patients.