First Personalized Gene Therapy
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genetic editing therapiesScience News
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Personalized gene editing saved a baby, but the tech’s future is uncertain
81% Informative
Baby KJ Muldoon has a disorder that prevents his liver from converting ammonia from broken-up proteins to urea.
KJ ’s form of the disease stems from a mutation in both copies of his CPS1 gene.
Without the enzyme, ammonia levels shoot up and can cause brain and nerve damage and death.
KJ got his first intravenous infusion containing the therapy in February .
He got a very low dose to start with. Two subsequent doses have been higher.
KJ may soon be able to leave the hospital where he has lived since birth.
There are more than 7,000 known genetic diseases. 15 to 20 percent of those might be fixable using currently available gene-editing technology.
Funds have been a common and concerning theme for gene therapy.
Companies often don’t have the resources to conduct clinical trials to get FDA approval.
Drastic cuts in research funding may also hinder early gene therapy development in academic labs.
VR Score
82
Informative language
82
Neutral language
47
Article tone
semi-formal
Language
English
Language complexity
53
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Attention-grabbing headline
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Known propaganda techniques
not detected
Time-value
long-living
External references
2
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2
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