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however, we included a no-template (ddHO) control during PCR to monitor potential contamination

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As research progresses, scientists are working to refine delivery methods, improve enzyme stability and determine how well the approach works across different ages and hair types

Track and follow up for adherence, AE reporting, and adjustments

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(2020) An Introduction to Gene Therapy for Genetic Diseases by BluebirdBio Autologous transplants, using the patients own stem cells does not need HLA matching and so is potentially available to all patients and so risks are reduced