Gene therapy
Gene therapy uses molecular tools to change the genetic code in cells and correct DNA mutations with the goal of curing diseases, including cancers and inherited genetic disorders such as hemophilia and sickle cell disease.
Here you can learn about how it works, the companies developing gene therapies, and the debate about the cost of these treatments.
Latest
STAT Plus: New gene therapy, to be priced at $4.25 million, has already transformed children’s lives
STAT Plus: FDA approves first gene therapy for fatal neuron disease in children
Listen: A dilemma in ALS, the first MASH drug, & why gene therapy is hard
STAT Plus: Hemophilia gene therapies arrived after 40 years of struggle. Where are the patients?
Meet the fetal surgeon forging CRISPR’s next frontier: curing diseases in the womb
STAT Plus: Ring Therapeutics, a Flagship gene therapy startup, lays off 20% of staff
STAT Plus: A gene therapy designed to heal skin wounds helped preserve a boy’s vision
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