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- W2894906221 abstract "In this commentary, we raise concerns about the compassionate use of CRISPR-mediated gene therapies in pediatric and perinatal patients. There is already a precedent for obtaining gene therapies for pediatric patients through compassionate use programs, and the recent passage of a federal Right to Try law may contribute to an increase in the number of patients who seek access to investigational products outside of a clinical trial. Clinicians, nurses, drug companies, and parents need support as they grapple with whether compassionate use of CRISPR-mediated gene therapies is the right thing to pursue for a child. We raise three issues to consider in that decision: (1) the effects of compassionate use on scientific research; (2) hype and harms of gene therapies; and (3) the limits and scope of parental authority." @default.
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- W2894906221 date "2018-12-01" @default.
- W2894906221 modified "2023-10-16" @default.
- W2894906221 title "Compassionate use of gene therapies in pediatrics: An ethical analysis" @default.
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- W2894906221 doi "https://doi.org/10.1053/j.semperi.2018.09.010" @default.
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