FDA approval: The biggest obstacle before the widespread use of gene therapy in the USA is obtaining FDA approval. A phase 3 trial is required prior to approval, treatment must show efficacy and minimal side effects compared to the disease being treated. Phase 3 trials have had problems with immune system rejection of the wrapping material/virus for the gene therapy as well as issues with dosing..
Answered 10/22/2017
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2 doctors weighed in across 2 answers
3 doctors weighed in across 2 answers
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