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A Modeling Study of Potential Impact of Gene Correction in Early Development on Severe Monogenic Diseases in the US

2025-11-21

Abstract excerpt

Pre-birth gene correction offers a precise and potentially transformative strategy to prevent severe genetic diseases by repairing pathogenic variants in embryos or gametes. Unlike selection-based preimplantation genetic testing (PGT), which depends on the availability of unaffected embryos, pre-birth gene correction directly addresses the underlying mutation, expanding the number of viable embryos available for t...

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Literature Corpus work
c16d5a94-ca45-5161-877e-1d8b81957994
DOI
10.20944/preprints202511.1543.v1
Open publication

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A Modeling Study of Potential Impact of Gene Correction in Early Development on Severe Monogenic Diseases in the USDOI 10.20944/preprints202511.1543.v1
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